neurology / en Our Commitment to Advancing Treatments for TK2d /solutions/magazine/detail/article/our-commitment-to-advancing-treatments-for-tk2d <span>Our Commitment to Advancing Treatments for TK2d</span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Fabian Somers, Patient Evidence </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2025-03-18T08:47:15+01:00" title="Tuesday 18 March 2025 - 08:47">Tue 18/03/2025 - 08:47</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2025-03/userphoto_6.jpg.webp?itok=OkXr6Vis" width="100" height="130" alt typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p><br>At ½šŗĢÓéĄÖ³Ē we are driven by our commitment to people living with severe diseases who inspire our research and development across neurology, immunology, and other areas where our, expertise, innovation and ambition align with unmet needs. &nbsp;</p><p>One such area of unmet need is in the management of thymidine kinase 2 deficiency (TK2d), where there are no approved therapies or international clinical guidelines. This rare, debilitating mitochondrial disorder affects children and adults, leading to progressive muscle weakness and severe mobility and respiratory challenges.</p><p>We recognize the urgent need for innovative therapies and are committed to driving research forward. Our team is working tirelessly to advance potential treatments that target the underlying causes of TK2d, offering hope to patients and families who currently have limited options. By collaborating with leading researchers, patient advocacy groups, and the medical community, we aim to accelerate the development of effective treatments that improve quality of life.</p><p>Every breakthrough begins with a commitment to those affected. We stand with the TK2d community and will continue to invest in advancing medical education and breakthrough science, ensuring that no one facing this condition is left without hope.</p><p>Stay connected with us for updates on our progress and learn how weā€™re making a difference. Together, we can bring new possibilities to those in need.&nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1909" hreflang="en">solutions</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/10191" hreflang="en">TK2d</a> <a href="/taxonomy/term/1895" hreflang="en"> Rare Disease</a> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15471&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="QeXa6piQQdRXhzxJhzNaFGHcRVAK-jEjJphWKxQ3eqY"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/solutions/magazine/detail/article/our-commitment-to-advancing-treatments-for-tk2d" data-a2a-title="Our Commitment to Advancing Treatments for TK2d"><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Fsolutions%2Fmagazine%2Fdetail%2Farticle%2Four-commitment-to-advancing-treatments-for-tk2d&amp;title=Our%20Commitment%20to%20Advancing%20Treatments%20for%20TK2d"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTQ3MSIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiI2MSIsImRpc2xpa2VzIjoiMCJ9"></a> <span class="like-15471"> 61 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Tue, 18 Mar 2025 07:47:15 +0000 Andrea_Puletto 15471 at More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies /solutions/magazine/detail/article/more-than-you-can-imagine-shedding-light-on-the-burden-of-rare-epilepsies <span>More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies </span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Andrea Wilkinson, Global Epilepsy &amp; Rare Syndromes </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2025-02-10T10:50:16+01:00" title="Monday 10 February 2025 - 10:50">Mon 10/02/2025 - 10:50</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2023-10/Andrea%20Wilkinson.png.webp?itok=DUxnwTL9" width="100" height="100" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p><br>When we think of epilepsies, the most common thing that comes to mind is seizures. While seizures remain the hallmark symptom of DEEs (Developmental and Epileptic Encephalopathy) such as Dravet syndrome, Lennox-Gastaut syndrome and CDKL5ā€Ædeficiency disorder, patients live with additional comorbidities that often go unnoticed but which have a profound and multifaceted impact on their lives. These rare conditions are associated with a host of physical, cognitive and behavioral challenges, taking a significant toll not only on individuals but also on caregivers and families, shaping every aspect of their daily lives.</p><p>With International Epilepsy Day on 10 February 2025, followed closely by Rare Disease Day on 28 February, themed <em>ā€˜More than you can imagineā€™</em>, itā€™s an opportune time to shine a light on the non-seizure burdens that these conditions are accompanied by, as well as the impact they have on both patients and their loved ones. &nbsp;</p><h3>Looking beyond seizures</h3><p><a href="/solutions/diseases/dravet-syndrome">Dravet syndrome (DS)</a>, <a href="/solutions/diseases/LGS">Lennox-Gastaut syndrome (LGS)</a> and <a href="/solutions/diseases/CDKL5-deficiency-disorder">CDKL5ā€Ædeficiency disorder (CDD)</a> are all rare forms of epilepsy classified as developmental and epileptic encephalopathies (DEEs). Living with these syndromes or conditions is far more complex than managing seizures alone, involving comorbidities that impact cognitive, emotional, and physical well-being. For instance, people with DS often contend with developmental delays, communication difficulties, and mobility issues such as a crouched gait.1 LGS is associated with profound intellectual disabilities and severe behavioural challenges such as aggression or hyperactivity, and CDD can result in musculoskeletal problems like scoliosis, lack of ability to develop speech, and delays or failure to achieve both gross and fine motor skills.</p><p>These DEEs donā€™t end with the individual ā€” they affect the quality of life of families and siblings, caregivers and their broader support system, causing a ripple effect that touches every facet of living.</p><p>Anxiety, constant vigilance, chronic sleep disruptions caused by night-time seizures, and the strain of navigating complex healthcare systems can leave families feeling overwhelmed. Caregivers often report high levels of emotional stress and information overload, particularly following diagnosis when their need for specialty healthcare and support is especially high. Furthermore, the unrelenting nature of evolving symptoms, compounded by frequent medical appointments and therapy sessions, can make day-to-day life exhausting and isolating.</p><p>Although peopleā€™s experiences of living with a DEE will be unique, there are some commonalities that may help to direct future research pursuits. For example, unpredictable seizure activity, irregular sleep patterns and disrupted behavior have all been reported by caregivers as negatively impacting quality of life, leading their loved ones to temporarily lose communication abilities or other activities of daily living. Gaining a better understanding of the disruptive nature of rare epilepsies, and the symptoms that matter most to patients and their families, will help researchers to prioritize areas of scientific focus and improve outcomes.</p><h3>Working together to address the non-seizure burden&nbsp;</h3><p>At ½šŗĢÓéĄÖ³Ē, we firmly believe that improving care for people living with epilepsy means addressing both seizure and non-seizure-related outcomes as well as survival of these complex conditions. This requires a holistic approach, with research that expands on current disease understanding, combined with initiatives that promote earlier diagnosis,ā€Æenhance treatment innovation, and strengthen support systems. Together, we must foster greater recognition of rare epilepsies, prioritizing both medical and non-medical needs to enhance both patient and family outcomes.&nbsp;</p><h3>ā€˜More Than You Can Imagineā€™ ā€“ A theme to remember&nbsp;</h3><p>The 2025 theme of Rare Disease Day is a timely reminder that while rare conditions like DS, LGS and CDD may be classified under the umbrella of ā€œepilepsyā€, their complexities extend far beyond seizures. Recognizing these challenges ā€“ and addressing them ā€“ could change what is often an overwhelming diagnosis into a life supported by compassionate resources and initiatives. &nbsp;</p><p>We invite you to help us increase understanding of the broader impacts of epilepsy. Whether itā€™s by engaging with advocacy organizations, supporting caregivers, or simply listening to the experiences of those affected, we can all contribute to building a world where no one feels unsupported in their epilepsy journey. Because when you look beyond seizures, youā€™ll find stories of resilience, expert voices longing to be heard, and an urgent need to bridge gaps in treatment and care. Together, we can inspire more comprehensive care models that look beyond seizures to encompass the broader spectrum of challenges that rare epilepsy communities face.&nbsp;</p><p>&nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1909" hreflang="en">solutions</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/10191" hreflang="en">TK2d</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15403&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="fooraoAvs8Jw5hOQ1ia6Eu5WKG4N_FzB9TnqQAKI5Bg"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/solutions/magazine/detail/article/more-than-you-can-imagine-shedding-light-on-the-burden-of-rare-epilepsies" data-a2a-title="More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies "><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Fsolutions%2Fmagazine%2Fdetail%2Farticle%2Fmore-than-you-can-imagine-shedding-light-on-the-burden-of-rare-epilepsies&amp;title=More%20Than%20You%20Can%20Imagine%3A%20Shedding%20Light%20on%20the%20Burden%20of%20Rare%20Epilepsies%20"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTQwMyIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiI2MCIsImRpc2xpa2VzIjoiMCJ9"></a> <span class="like-15403"> 60 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Mon, 10 Feb 2025 09:50:16 +0000 Andrea_Puletto 15403 at Advancements in Epilepsy Research /innovation/magazine/detail/article/advancements-in-epilepsy-research <span>Advancements in Epilepsy Research</span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Liesbeth FranƧois, Data and Translational Sciences </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2024-08-01T13:25:23+02:00" title="Thursday 1 August 2024 - 13:25">Thu 01/08/2024 - 13:25</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2024-08/userphoto_3.jpg.webp?itok=R7ptMoUN" width="72" height="79" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p><br>Although the field of epilepsy research is well-established, we have only just begun to scratch the surface of our understanding of more complex epilepsies. We are entering a period of disruption, where new breakthroughs and the integration of innovative technologies into drug discovery and development show promise in transforming epilepsy care. One area is ā€˜<strong>molecular hallmarks</strong>ā€™ ā€“ molecular signatures that contribute to the presentation of complex epilepsies.&nbsp;</p><h2>Discovering ā€˜molecular hallmarksā€™&nbsp;</h2><p>At ½šŗĢÓéĄÖ³Ē, a key area of our research is focused on broadening targets for treatments. In a study published earlier this year, we identified well-known and new gene regulatory networks shared across different refractory epilepsies; we called these ā€˜molecular hallmarksā€™.i This research was the culmination of five years of collaborative work that has enabled us to start to advance our understanding of hard-to-treat epilepsies and opened new possibilities for drug-target discovery and development. &nbsp;</p><p>ā€˜Molecular hallmarksā€™ are currently under study by our Early Development team who have mapped out a strategy for further research to potentially make the hallmarks targetable. The research targets include neuronal support and myelination, neuroinflammation and immune response, energy metabolism, and the brain extracellular matrix (ECM). &nbsp;</p><h2>Targeting complex epilepsies&nbsp;</h2><p>Epilepsy is more than seizures and we are continuing to uncover the vast complexities of the condition. ā€˜Molecular hallmarksā€™, therefore, offer potential additional targets for epilepsy treatments that go beyond seizure suppression, enabling us to target the underlying mechanisms of the most challenging seizure disorders. This shift represents a move away from conventional treatment methods, and into research that focuses potentially disease modifying therapies.</p><p>This ā€˜molecular hallmarkā€™ research is an important part of our epilepsy strategy and provides a data-driven framework for the further identification and intervention of further dysregulated biological pathways.</p><p>Ultimately, our goal at ½šŗĢÓéĄÖ³Ē is to develop new treatment options, based on these scientific discoveries, that can attempt to enhance patient outcomes. &nbsp;</p><h2>The role of computational biology &nbsp;</h2><p>Epilepsy is not a one-size-fits-all condition. Each personā€™s experience of living with epilepsy is unique and given what we know so far about the complexity of epilepsies, there is a clear role for computational biology in epilepsy research. Through analysis of the expression profiles of refractory epilepsies, we can start to understand global biological mechanisms that are impacted in refractory epilepsies. Our research continues to drive our understanding of the human pathobiology of different refractory epilepsies to support our aim to identify new disease modifying treatments for patients. This research aims to help us comprehend the mechanisms underpinning epilepsy and enable the development of new therapies for refractory epilepsies. &nbsp;</p><h2>The future of research&nbsp;</h2><p>Everything we do starts with people and families living with epilepsies, helping them achieve their ideal and maximize their life opportunities. Looking to the future as we advance our epilepsy research, we recognize that those living with complex epilepsies will continue to rely on chronic, symptomatic anti-seizure medicines, and we continue our research to help improve outcomes with the symptomatic approaches available to patients today. However, we constantly need to push our approaches to secure the best outcome for those we serve. Molecular hallmarks and master regulators provide invaluable insights and open new avenues for precision medicine and new treatments that address the unmet needs of people living with epilepsies.&nbsp;</p><p>&nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1910" hreflang="en">innovation</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> <a href="/taxonomy/term/5431" hreflang="en"> Neurology &amp; Therapy</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15056&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="xX-ffr0c3k9PZAvR3kt7Jgy0uYgAJqjVRrtxW2dSPpk"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/innovation/magazine/detail/article/advancements-in-epilepsy-research" data-a2a-title="Advancements in Epilepsy Research"><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Finnovation%2Fmagazine%2Fdetail%2Farticle%2Fadvancements-in-epilepsy-research&amp;title=Advancements%20in%20Epilepsy%20Research"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTA1NiIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiIyMzMiLCJkaXNsaWtlcyI6IjMifQ%3D%3D"></a> <span class="like-15056"> 233 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Thu, 01 Aug 2024 11:25:23 +0000 Andrea_Puletto 15056 at Supporting International CDKL5 Day /about-ucb/magazine/detail/article/supporting-international-cdkl5-day <span>Supporting International CDKL5 Day </span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Nick Francis, Neurology Communication </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2024-06-17T11:13:13+02:00" title="Monday 17 June 2024 - 11:13">Mon 17/06/2024 - 11:13</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/author_images/Nick_Francis.jpg.webp?itok=zF6BqdO1" width="50" height="66" alt="Picture of author Katrin Hoffmann" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p>&nbsp;</p><p>Every June the CDKL5 community comes together to recognize CDKL5 disorder, and promote awareness efforts, research, collaboration, and fundraising. &nbsp;</p><p>June 17 is International CDKL5 Day, in memory of Glyn Boltwood, whose life and legacy was directly responsible for the medical and scientific community discovering CDKL5 Disorder in 2004.</p><p>CDKL5 deficiency disorder ā€“ a Developmental and Epileptic Encephalopathy (DEE) - is a genetic disorder that causes seizures, developmental delay, and severe intellectual disability. Seizures typically begin within a few months after birth and are difficult to control with medications. Most children have 1 to 5 seizures every day. Other symptoms include problems with sleeping, feeding, and teeth grinding. Gastrointestinal symptoms are also common and may include constipation, reflux, and air swallowing. &nbsp;</p><p>About 1 in 5 children use a feeding tube. CDKL5 deficiency disorder occurs more often in females than males, and males usually have more severe symptoms. This disorder is caused by a change (pathogenic variant) in the CDKL5 gene that is usually not inherited from either parent (de novo). It is an X-linked dominant disorder. CDKL5 deficiency disorder was once thought to be a variant of Rett syndrome but is now considered a separate disorder.&nbsp;</p><p>For more useful resources and information, visit:</p><p><a href="https://rarediseases.info.nih.gov/diseases/12173/cdkl5-deficiency-disorder ">https://rarediseases.info.nih.gov/diseases/12173/cdkl5-deficiency-disorder&nbsp;</a></p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1907" hreflang="en">about ucb</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> <a href="/taxonomy/term/10345" hreflang="en">CDKL5</a> <a href="/taxonomy/term/1617" hreflang="en"> seizures</a> <a href="/taxonomy/term/10503" hreflang="en">Developmental and Epileptic Encephalopathy</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15037&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="IjTgXFFU40XJZfStTEw7kEOZugTEr7EmaX-pQStRgog"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/about-ucb/magazine/detail/article/supporting-international-cdkl5-day" data-a2a-title="Supporting International CDKL5 Day "><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Fabout-ucb%2Fmagazine%2Fdetail%2Farticle%2Fsupporting-international-cdkl5-day&amp;title=Supporting%20International%20CDKL5%20Day%20"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTAzNyIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiIyMTciLCJkaXNsaWtlcyI6Ii0yIn0%3D"></a> <span class="like-15037"> 217 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Mon, 17 Jun 2024 09:13:13 +0000 Andrea_Puletto 15037 at Innovating in Epilepsy /innovation/magazine/detail/article/innovating-in-epilepsy <span>Innovating in Epilepsy </span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Mike Davis, Epilepsy &amp; Rare Syndromes </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2024-06-14T13:19:04+02:00" title="Friday 14 June 2024 - 13:19">Fri 14/06/2024 - 13:19</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2022-10/Mike%20Davis.jpg.webp?itok=jKOt-5dM" width="84" height="100" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p>&nbsp;</p><p>In recent years, epilepsy research has evolved from focusing solely on symptomatic control of seizures to investigating the underlying pathologies, aiming to develop disease-modifying and ultimately curative therapies.</p><p>At ½šŗĢÓéĄÖ³Ē, we understand the importance of staying at the forefront of innovation, and prioritizing research and development within the dynamic category of epilepsy - principles that have built our 30-year heritage in this area. To ensure we are a part of this evolution, we need to advance and progress. Our people-first approach focuses on three areas of development: &nbsp;</p><ol><li>Driving innovation in science &nbsp;</li><li>Partnering with patients to advance the ecosystem &nbsp;</li><li>Elevating the standard of care for all people living with epilepsies &nbsp;</li></ol><p>We are entering a rapid period of disruption where we need to address multiple unmet needs of specific patient populations through evolving modalities, targets, and care delivery. To achieve this, we need to adopt new approaches that employ emerging technologies and require capabilities beyond anything currently present in the market. &nbsp;</p><p>We are focused on the development of an extensive future epilepsy portfolio, made up of targeted, precision medicines, focusing on the entire spectrum of epilepsies. As well as continually improving the outcomes of the symptomatic approaches, available to patients today.&nbsp;</p><h2>Advancing symptomatic care &nbsp;</h2><p>Our research focuses on developing a better understanding of the prevention of seizures and the underlying mechanisms of the most challenging seizure disorders. We are advancing distinct solutions across many different epilepsies for specific patient populations, and have several early discovery phase research projects, involving both small molecules and gene therapies, that are currently active. &nbsp;</p><p>Our goal is to disrupt this space by advancing solutions across many different domains and dimensions of the epilepsies. This starts with our current products where we continue to research their impact on non-seizure outcomes relating to cognition, behavior, emotional development, and sleep architecture; neurodevelopment delays; corollary links to other neurodegenerative diseases; Sudden Unexpected Death in Epilepsy (SUDEP) and all-cause mortality.&nbsp;</p><h2>Disease modification&nbsp;</h2><p>Teams from across ½šŗĢÓéĄÖ³Ē are envisioning a next generation of therapies that will take epilepsy care beyond symptomatic treatments by targeting the underlying mechanisms that lead to both seizure and non-seizure effects of the diseases and syndromes we aim to treat and opening the door to disease modifying and curative solutions. Working together, we are exploring fundamental disease etiologies across the epilepsies, uncovering root causes of monogenic epilepsies, and targeting the pathobiology of structural epilepsies, such as the role neuronal support and myelination, neuroinflammation and immune response, energy metabolism, and the brain extracellular matrix (ECM) play in the refractory complex epilepsies.</p><h2>The role of AI&nbsp;</h2><p>Technology and AI are having an increasingly significant role in epilepsy research and development - for instance, to help patients and families better characterize and manage their seizures. Our technology-based partnerships with Eysz and NextSense introduce new ways to track brain activity outside traditional EEG monitoring. Furthermore, our R&amp;D teams continually harness ½šŗĢÓéĄÖ³Ēā€™s deep institutional knowledge of the pathobiology of epilepsies. &nbsp;Using advanced analytics, we leverage AI resources to strengthen our discovery and development efforts. This includes identifying mechanisms and developing disease models through to prioritizing potential targets.</p><h2>Broadening access&nbsp;</h2><p>At ½šŗĢÓéĄÖ³Ē, we believe that all patients who need our treatments should have access to them now and in the future. Health inequities are a challenge in bringing innovations to people living with epilepsies and elevating their standard of care. Weā€™re working to address this through sustainable approaches to reach the historically underserved people of lower-middle-income countries and address their unmet needs. For instance, in Rwanda, we have recently worked with the government to enable those living with epilepsy to have access to ½šŗĢÓéĄÖ³Ē treatment. &nbsp;</p><p>Health equity is also about deepening our local connections in the communities where we operate. This starts with understanding how diverse patient groups experience care and treatment, to better understand where social determinants of health may have exacerbated health inequities among historically underserved communities and define targeted approaches.&nbsp;</p><h2>Future thinking&nbsp;</h2><p>Looking ahead, we expect to see the continuing emergence of treatment modalities, delivery technologies, and hidden insights into underlying pathobiology. This has the potential to advance the development of disease-modifying solutions and support the progress toward curative medicine options for those living with epilepsies. Ultimately, our goal at ½šŗĢÓéĄÖ³Ē is to continue to innovate across the entire spectrum of epilepsy care ā€“ we want to treat the treatable and cure the curable.</p><p>&nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1910" hreflang="en">innovation</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/5431" hreflang="en"> Neurology &amp; Therapy</a> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15035&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="CLb4h5pCOL10E5QqKPVBJnpGhYhoMAWk353to2NKu-8"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/innovation/magazine/detail/article/innovating-in-epilepsy" data-a2a-title="Innovating in Epilepsy "><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Finnovation%2Fmagazine%2Fdetail%2Farticle%2Finnovating-in-epilepsy&amp;title=Innovating%20in%20Epilepsy%20"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTAzNSIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiIyMzgiLCJkaXNsaWtlcyI6Ii00In0%3D"></a> <span class="like-15035"> 238 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Fri, 14 Jun 2024 11:19:04 +0000 Andrea_Puletto 15035 at Making a difference together - First ½šŗĢÓéĄÖ³Ē Pan-European Meeting on Developmental and Epileptic Encephalopathies (DEEs) /innovation/magazine/detail/article/making-a-difference-together-first-ucb-pan-european-meeting-on-developmental-and-epileptic-encephalopathies-dees <span>Making a difference together - First ½šŗĢÓéĄÖ³Ē Pan-European Meeting on Developmental and Epileptic Encephalopathies (DEEs)</span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Francesco Chiaromonte, HCP Engagement </div> <span><span lang about="/user/9417" typeof="schema:Person" property="schema:name" datatype>Andrea_Puletto</span></span> <span><time datetime="2024-05-17T16:14:07+02:00" title="Friday 17 May 2024 - 16:14">Fri 17/05/2024 - 16:14</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2024-05/untitled_0.png.webp?itok=2wBUUSDS" width="200" height="200" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p>&nbsp;</p><p>At ½šŗĢÓéĄÖ³Ē, we believe that providing opportunities for collaboration, discussion, and knowledge exchange for healthcare professionals can help make a real difference in improving the care and treatment of people living with serious neurological conditions, such as epilepsy. For this reason, we recently launched a new European meeting series for healthcare professionals, entitled: "Make the difference together". &nbsp;</p><p>The first two-day meeting in this series took place in March, with more than 35 Neurologists and Neuropaediatricians from across Europe in attendance. The topic of discussion was Developmental and Epileptic Encephalopathies (DEEs) ā€“ a key area of focus for ½šŗĢÓéĄÖ³Ē. &nbsp;</p><p>On the first day, delegates highlighted the importance of a patient-centered approach to the management of DEEs, in addition to advances in research and their impact on clinical practice. Following this, clinical case studies of people living with Lennox-Gastaut syndrome were discussed in small groups; the various cases showing just how complex these diseases are. The intensive and practical exchange among the participants resulted in many hands-on considerations on how to improve care.</p><p>The second day of the meeting began with a discussion about the opportunities and challenges of transitioning patients from pediatric to adult care. Discussion then turned to the topic of optimizing the management of DEEs. Emphasis was placed on the importance of individual action plans and prioritizing families, who often take on a large share of home care. The event concluded with a group discussion of complex clinical case studies of people living with Dravet syndrome. The results of these constructive and practice-oriented group discussions were then compiled and discussed again in the plenary session. &nbsp;</p><p>Our first pan-European meeting "Make the difference together" confirmed once again that these deep discussions are extremely valuable in helping improve patient care, and the positivity of the meeting was reflected in the voices of the participants. &nbsp;</p><p>We would like to thank all the participating experts for their contributions, which greatly enriched the event, and we thank them for their commitment to people living with epilepsy.</p><p>The next event will take place in June (21-22), in Prague. Do please get in touch if you want further information.</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1910" hreflang="en">innovation</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/10486" hreflang="en">Pan-European Meeting on Developmental and Epileptic Encephalopathies DEEs</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=15015&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="KbbT07lrqJenFiGevWhQ6Mu6C6FCohEE1ZxPRkCqsSg"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> <div class="comment-wrap" data-comment-user-id="0" id="comment-43685"> <span class="block">Comment:</span> <div id="comment-43685" class="comment"> <span class="name-date"> Posted by <strong><span lang typeof="schema:Person" property="schema:name" datatype>Heidi Henninger </span></strong>, 21 May 2024 </span> <div class="comment--body"> <div class="field field--name-comment-body field--type-text-long field--label-hidden field__items"> <p>Thank you ½šŗĢÓéĄÖ³Ē for your leadership in this important area. Let us continue to move forward together!</p> </div> </div> </div> <drupal-render-placeholder callback="comment.lazy_builders:renderLinks" arguments="0=43685&amp;1=default&amp;2=en&amp;3=" token="43njaomFWCNvp_RXvvztAgo6k6tqOpSPrD97GvWpSoI"></drupal-render-placeholder> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/innovation/magazine/detail/article/making-a-difference-together-first-ucb-pan-european-meeting-on-developmental-and-epileptic-encephalopathies-dees" data-a2a-title="Making a difference together - First ½šŗĢÓéĄÖ³Ē Pan-European Meeting on Developmental and Epileptic Encephalopathies (DEEs)"><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Finnovation%2Fmagazine%2Fdetail%2Farticle%2Fmaking-a-difference-together-first-ucb-pan-european-meeting-on-developmental-and-epileptic-encephalopathies-dees&amp;title=Making%20a%20difference%20together%20-%20First%20½šŗĢÓéĄÖ³Ē%20Pan-European%20Meeting%20on%20Developmental%20and%20Epileptic%20Encephalopathies%20%28DEEs%29"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNTAxNSIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiIzMTUiLCJkaXNsaWtlcyI6IjIifQ%3D%3D"></a> <span class="like-15015"> 315 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Fri, 17 May 2024 14:14:07 +0000 Andrea_Puletto 15015 at New approaches in epilepsy research /about-ucb/magazine/detail/article/new-approaches-in-epilepsy-research <span>New approaches in epilepsy research</span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Stefanie Dedeurwaerdere, Epilepsy Discovery Research </div> <span><span lang about="/user/8781" typeof="schema:Person" property="schema:name" datatype content="Vlastimil.Vanco@ucb.com">Vanco Vlastimiā€¦</span></span> <span><time datetime="2023-06-13T13:01:22+02:00" title="Tuesday 13 June 2023 - 13:01">Tue 13/06/2023 - 13:01</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2023-06/Stefanie%20Dedeurwaerdere_profile.jpg.webp?itok=avsFkbNz" width="88" height="100" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p><br> At ½šŗĢÓéĄÖ³Ē, we have been leaders in the research and development of treatments for epileptic seizures for more than 20 years. In 2022, more than 2.6 million people living with epilepsy benefited from our anti-seizure medications, and our commitment to providing new solutions for those in need continues. We are working towards a future where we hope to impact the underlying causes of the epilepsies with disease modifying therapies which can change the course of the disease.<br> <br> It is important to recognise that there are multiple types of epilepsy. In fact, we now more commonly refer to epilepsy as the ā€˜epilepsies,ā€™ indicating that itā€™s not really a single disease. In addition, for many people ā€” particularly those living with certain, rare epileptic syndromes ā€” seizures are not the only impactful symptom.<br> <br> As an industry, we've been very focused on seizures, which for so many patients, continues to be top of mind. But itā€™s essential that, in our focus on seizure suppression, we donā€™t neglect what changes in the brain are often underneath those seizures.<br> <br> When we understand the root cause of certain epilepsies, it opens opportunities to target the underlying mechanisms that impact these diseases, through new treatment approaches.&nbsp;<br> <br> To further increase our understanding, we've been collaborating with universities on building one of the largest analyses of human brain tissues from epilepsy patients in the world, including &gt; 200 samples. This in-depth study allows us to really comprehend the world of epilepsies as a global brain map, to quantify fully the different subtypes of epilepsy, understanding what they may have in common and what could be unique to specific conditions.&nbsp;<br> <br> For complex epilepsies, where mechanisms are less understood, the situation is more challenging. Here, we are building on genetics to help understand universal disease mechanisms across the epilepsies.<br> <br> In addition, we have also developed an artificial intelligence computational framework in collaboration with various academic partners to investigate disease mechanisms and processes that helps us to identify novel areas for future therapeutic entry points.&nbsp;<br> <br> Our digital innovations are also key in allowing our patients to have a louder voice, to feel more empowered, and to know and understand their disease more thoroughly. The combination of more diverse treatment options and a more informed patient population will help bring us in an era where the treatments will be much more personalized, impacting patientsā€™ lives in a more significant way.&nbsp;<br> <br> Our commitment to epilepsy research has never been stronger, and as the science advances so does our curiosity. We are hopeful for the future, and we will learn and refine our thinking as we progress.<br> &nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1907" hreflang="en">about ucb</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> <a href="/taxonomy/term/1266" hreflang="en">patients</a> <a href="/taxonomy/term/1647" hreflang="en"> research </a> <a href="/taxonomy/term/1352" hreflang="en"> innovation </a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=14835&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="QrknJdyuvIT7CZ3uIh_X2uVYNVecsn1US8JEZZKvHw0"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/about-ucb/magazine/detail/article/new-approaches-in-epilepsy-research" data-a2a-title="New approaches in epilepsy research"><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Fabout-ucb%2Fmagazine%2Fdetail%2Farticle%2Fnew-approaches-in-epilepsy-research&amp;title=New%20approaches%20in%20epilepsy%20research"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNDgzNSIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiI0OTQiLCJkaXNsaWtlcyI6Ii01NCJ9"></a> <span class="like-14835"> 494 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Tue, 13 Jun 2023 11:01:22 +0000 Vanco Vlastimil (½šŗĢÓéĄÖ³Ē S.A.) 14835 at ā€œDisease modificationā€ in epilepsies: key questions answered /innovation/magazine/detail/article/disease-modification-in-epilepsies-key-questions-answered <span>ā€œDisease modificationā€ in epilepsies: key questions answered</span> <div class="field field--name-field-newsroom-author-title field--type-string field--label-above field__items"> Dr Konrad Werhahn, Global Medical Affairs, Epilepsy &amp; Rare Syndromes </div> <span><span lang about="/user/9411" typeof="schema:Person" property="schema:name" datatype>Chiara Vona</span></span> <span><time datetime="2023-06-01T14:45:55+02:00" title="Thursday 1 June 2023 - 14:45">Thu 01/06/2023 - 14:45</time> </span> <div class="field field--name-field-newsroom-author-image field--type-entity-reference field--label-above field__items"> <article class="media media--type-image media--view-mode-default"> <div class="field field--name-image field--type-image field--label-hidden field__items"> <img loading="lazy" src="/sites/default/files/styles/ucb_header_image/public/2023-06/Dr%20Konrad%20Werhahn_res.jpg.webp?itok=IoWctR-8" width="85" height="85" typeof="foaf:Image"> </div> </article> </div> <div class="field field--name-field-newsroom-content field--type-text-long field--label-above field__items"> <p><br> Epilepsy research is entering an exciting new chapter, with researchers delving deeper into the concept of ā€œdisease modificationā€ - aiming to develop treatments that not only better control symptoms, but also may modify the underlying disease process.&nbsp;<br> <br> While the potential of disease modification in epilepsy treatment is promising, several challenges need to be addressed. These include identifying appropriate therapeutic targets, optimizing drug delivery methods, ensuring safety and efficacy, and conducting robust clinical trials to establish the long-term benefits of disease-modifying treatments.&nbsp;<br> <br> As we have seen in other therapeutic areas such as Alzheimerā€™s disease or Parkinsonā€™s disease, as the science advances it will become increasingly important for the pharmaceutical industry, regulators, healthcare professionals, and patient community, to agree on a more accurate definition of what is meant by ā€œdisease modificationā€ in any given situation so that the effect and outcomes of any potential new treatments can be accurately measured and interpreted.<br> <br> While still in the early stages of development, the progress being made in understanding the molecular and genetic basis of epilepsy is bringing us closer to the realization of disease-modifying treatments, offering hope for a brighter future for individuals living with epilepsy.<br> <br> <strong>What do we mean by ā€œdisease modificationā€ in epilepsy?</strong><br> <br> ā€œDisease modificationā€ describes changes that affect the underlying pathophysiology of a disease and have beneficial outcome on the clinical course. In simple terms, this means treating the root cause of disease, rather than addressing individual symptoms. This is a descriptive term, not a single well-defined effect. It requires careful interpretation within a given therapy area and disease, and it may take many forms with several distinct outcomes based on the nature of the epileptic condition. This means that our job in epilepsies is to clarify exactly what kind of disease-modifying effect we are seeking in different types of epilepsy, and how this is best measured.<br> <br> <strong>Where we are now?</strong><br> <br> To date, treatment of epileptic seizures has been based on addressing symptoms, with medication targeted at raising the threshold to the occurrence of epileptic seizures. While this helps address disruption and potential damage caused by a symptom (seizures), it does not significantly impact comorbidities such as learning and behavior, which are particularly important because epilepsies frequently begin in childhood.<br> <br> At the other end of the spectrum, a curative treatment is specific to the disease. While symptomatic treatments may work across several diseases with the same symptom(s), curative treatments work to correct underlying pathobiology ā€“ the biological root of disease. This approach addresses co-morbidities beyond the primary symptom, with the possible benefit that it may result in a longer-lasting effect than symptom-based treatment.<br> <br> <strong>How are we approaching disease modification in research and development and the clinic?</strong><br> <br> Research into disease modification and identification of disease-modifying treatment targets is approached from two perspectives:<br> ā€¢&nbsp;&nbsp; &nbsp;Outside-in: developing our knowledge of the natural history of disease, understanding non-seizure symptoms and outcomes&nbsp;<br> ā€¢&nbsp;&nbsp; &nbsp;Inside-out: understanding of biological pathways and identifying targets as close to the root cause of the disease as possible<br> <br> Through this combination of careful observation of the whole symptom picture and understanding of how epilepsies progress, coupled with research into the pathobiology of disease, we hope to identify suitable targets for disease modification.<br> <br> <strong>What should disease-modification-based treatment goals look like in epilepsies?</strong><br> <br> Disease-modifying treatments will change how we approach the care of patients with epilepsies, but they will also require new approaches to constructing and measuring the success of clinical trials.</p> <p>Conventional design for clinical studies can fall short when applied to disease-modifying treatments, due to reliance on narrow, short-term outcome measures, inappropriate efficacy endpoints, and potentially unacceptably high risks to patients with refractory epilepsy, high seizure burdens, and elevated baseline risk of sudden unexplained death in epilepsy patients (SUDEP). &nbsp;Suitable replacements for traditional endpoints may include markers of developmental achievement, language and communication, sleep, behavior, and motor function.</p> <p>As we look to evolve our approach to the treatment of epilepsies, another challenge is regulatorsā€™ inclination to be specific and pragmatic, which leads us towards defining treatment by ā€œwhat the drug does.ā€ This has an inherent bias towards a focus on symptoms. However, study design and treatment labelling in oncology and (closer to our neurobiological home) in Alzheimerā€™s disease and multiple sclerosis have established precedents in terms of defining efficacy through impact on disease progression. While there is no regulatory definition of disease modification for epilepsies, there is plenty of cause for optimism.<br> &nbsp;</p> </div> <div class="field field--name-field-newsroom-category field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/1910" hreflang="en">innovation</a> </div> <div class="field field--name-field-newsroom-tags field--type-entity-reference field--label-above field__items"> <a href="/taxonomy/term/5437" hreflang="en"> epilepsy</a> <a href="/taxonomy/term/1347" hreflang="en"> neurology</a> </div> <div> <div class="comments-wrapper"> <div class="comment-area"> <h2 class="red">Leave a Comment</h2> <drupal-render-placeholder callback="comment.lazy_builders:renderForm" arguments="0=node&amp;1=14830&amp;2=field_newsroom_askexpert&amp;3=ask_expert" token="qo9WI_ktJRx-4kirFY8SK8IEX2e84zUdqcAeqtXBLus"></drupal-render-placeholder> <span class="toggle-form js-toggle-form"></span> </div> </div> </div> <span class="a2a_kit a2a_kit_size_16 addtoany_list" data-a2a-url="/innovation/magazine/detail/article/disease-modification-in-epilepsies-key-questions-answered" data-a2a-title="ā€œDisease modificationā€ in epilepsies: key questions answered"><a class="a2a_dd addtoany_share" href="https://www.addtoany.com/share#url=https%3A%2F%2Fwww.ucb.com%2Finnovation%2Fmagazine%2Fdetail%2Farticle%2Fdisease-modification-in-epilepsies-key-questions-answered&amp;title=%E2%80%9CDisease%20modification%E2%80%9D%20in%20epilepsies%3A%20key%20questions%20answered"></a><a class="a2a_button a2a_button_facebook"><img src="/themes/custom/ucb_premier/images/a2a/facebook-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_linkedin"><img src="/themes/custom/ucb_premier/images/a2a/linkedin-icon.svg" width="16" height="16" border="0" alt="linkedin"></a><a class="a2a_button a2a_button_twitter"><img src="/themes/custom/ucb_premier/images/a2a/twitter-icon.svg" width="16" height="16" border="0" alt="twitter"></a></span> <div class="field field--name-field-like field--type-likes-dislikes field--label-above field__items"> <div class="like_dislike"> <div class="like"> <a rel="nofollow" class="use-ajax" href="/like-dislike/like/eyJlbnRpdHlfdHlwZSI6Im5vZGUiLCJlbnRpdHlfaWQiOiIxNDgzMCIsImZpZWxkX25hbWUiOiJmaWVsZF9saWtlIiwibGlrZXMiOiI0MTEiLCJkaXNsaWtlcyI6Ii0zOSJ9"></a> <span class="like-14830"> 411 Likes </span> </div> </div> <div id="like_dislike_status"></div> </div> Thu, 01 Jun 2023 12:45:55 +0000 Chiara Vona 14830 at