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Advancing neurodegenerative disease research at AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders

Andrea_Puletto — Wed, 02 Apr 2025 08:53:07 +0000

Advancing neurodegenerative disease research at AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders

Alexander Klein, Global Medical Neurodegeneration

Andrea_Puletto Wed 02/04/2025 - 10:53

At the AD/PD Congress, one of the leading global events for advancing science in Alzheimer’s and Parkinson’s diseases, we are reaffirming our commitment to tackling the challenges of these devastating neurodegenerative diseases. The conference provides an invaluable platform to connect with the scientific community, fuel dialogues that spark innovation, and drive progress in understanding the underlying mechanisms of these complex conditions.

Neurodegenerative diseases like Alzheimer’s and Parkinson’s present an urgent, unmet medical need. These conditions profoundly affect millions of individuals and their families worldwide, often robbing them of independence and quality of life. We recognize that current solutions often fall short in providing comprehensive care, and we are dedicated to researching to bridge these gaps with innovative solutions. To develop solutions effectively, we must first uncover the mysteries behind their onset and progression.

��ֳ�’s Parkinson's research program includes investigating mechanisms to inhibit the propagation of alpha-synuclein – a process believed to underlie the spread of neurodegeneration. Alongside this, we are advancing research into innovative therapies for symptom control, recognizing the diverse needs of each patient throughout their disease trajectory, from early to advanced stages.

We firmly believe that the patient voice is essential in shaping meaningful research and therapies. By prioritizing patient-led initiatives and collaborating directly with those affected, we ensure that our work reflects their needs, experiences, and hopes for the future. The Patient Engagement and Care Progression Research (PECPR) initiative, established in 2021 is a collaborative effort to bring the patient community into the heart of early research and clinical development. The initiative strives to improve patient outcomes by working together on a patient-centered research model, embedding patient involvement throughout ��ֳ�’s Parkinson’s drug development portfolio, and fostering global collaboration to advance Parkinson’s research.

In Alzheimer's, we're hoping to find therapies that can slow the progression of the disease, and recent data from our development program is also on show at AD/PD. Alzheimer’s disease occurs when harmful plaques and tangles comprised of β-amyloid and tau protein build up in the brain, disrupting its function and leading to cognitive decline and dementia.

By progressing the science, working together and listening to the patient voice, we aim to move closer to a world where no family has to face the burden of neurodegenerative disease alone. To every scientist and researcher who joined us at AD/PD — thank you. Your dedication and ideas inspire hope and progress. Together, we are working towards a better future in neurodegenerative science, for patients now and in the future.

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Innovating in neurology: ��ֳ�'s commitment shines at AAN 2025

Andrea_Puletto — Wed, 02 Apr 2025 08:31:25 +0000

Innovating in neurology: ��ֳ�'s commitment shines at AAN 2025

Donatello Crocetta, Global Rare Disease＆Rare Medical

Andrea_Puletto Wed 02/04/2025 - 10:31

As we prepare for the American Academy of Neurology (AAN) congress, we're excited to showcase ��ֳ�'s dedication to transforming care for individuals living with neurological conditions. The AAN annual meeting isn't just an event; it's a gathering of the brightest minds in neurology, where breakthroughs are shared, and challenges are tackled head-on.

A focus on valuable solutions

At ��ֳ�, our passion in neurology is deeply rooted in our history. We have always strived to push the boundaries and redefine what is possible in neurological care. From creating a meaningful impact for conditions like generalized Myasthenia Gravis (gMG) to developing solutions for rare epileptic syndromes such as Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), ��ֳ� has consistently led with innovation and compassion. Our research explores the effect of these conditions on quality of life, not just for patients but also for their families, paving the way for better disease management and, ultimately, better lives.

Showcasing innovation at AAN

Among the 24 abstracts accepted for the AAN congress, we are presenting new late-stage clinical trial findings that support the efficacy and safety of our therapies in epilepsy; insights from a new international patient registry for gMG; and findings that progress our understanding of the disease course of TK2d.

��ֳ� is proud to host its inaugural US Rare Disease Connect in Neurology (RDCN) Annual Summit at the 2025 American Academy of Neurology (AAN) Annual Meeting to provide a forum for needs-driven medical education for the generalized myasthenia gravis community (gMG). This event is being held exclusively for healthcare providers who are involved in patient care. RDCN is an ongoing global learning program connecting a community of experts committed to delivering excellence for people living with gMG. The RDCN network is expanding to the US, specifically with the goal to foster collaboration for comprehensive gMG care.

Our commitment to science and care

First and foremost, ��ֳ�’s presence at AAN reflects our unwavering commitment to collaboration with the scientific, medical, and patient communities. We believe that engaging directly with those who live with these conditions, their caregivers, and healthcare providers allows us to tailor our strategies and focus on what truly matters.

Our commitment to collaboration and leveraging real-world insights is central to our approach as we strive to create a future where every person with a neurological condition can achieve optimized care and management. Together, we can drive change and shape a brighter future in the field of neurology, ensuring that our efforts translate into meaningful improvements for those who rely on our innovations.

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innovation epilepsy neurology

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Our Commitment to Advancing Treatments for TK2d

Andrea_Puletto — Tue, 18 Mar 2025 07:47:15 +0000

Our Commitment to Advancing Treatments for TK2d

Fabian Somers, Patient Evidence

Andrea_Puletto Tue 18/03/2025 - 08:47

At ��ֳ� 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.

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.

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.

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.

Stay connected with us for updates on our progress and learn how we’re making a difference. Together, we can bring new possibilities to those in need.

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TK2d Rare Disease neurology

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Exploring the future of immuno-dermatology care: ��ֳ� at AAD Annual Meeting 2025

Andrea_Puletto — Fri, 07 Mar 2025 10:45:19 +0000

Exploring the future of immuno-dermatology care: ��ֳ� at AAD Annual Meeting 2025

Simon Lucas, Patient Evidence

Andrea_Puletto Fri 07/03/2025 - 11:45

This week for the 2025 Annual Meeting of the American Academy of Dermatology (AAD), we look forward to connecting with clinicians, researchers and the dermatology community in Orlando who share our passion for advancing patient care. This flagship event in the dermatology calendar promises to be a dynamic forum for advancing scientific exchange and fostering the connections that lead to life-changing solutions.

Chronic immuno-dermatology diseases place a significant burden on patients and while important advances have been made in recent years, we remain deeply committed to addressing the substantial unmet needs and improving the lives of those affected. Long-term data are particularly meaningful, providing deeper insights that are especially important for those living with chronic conditions.

This year, for AAD we are excited to continue to expand the growing body of research across multiple immuno-dermatology diseases with the presentation of 18 abstracts, including in hidradenitis suppurativa (HS) and psoriasis, as part of our ambitious research programs aimed at transforming the future care within the next decade.

Visitors to our exhibition booth will have the opportunity to learn about our ongoing research, patient support programs and education initiatives. We look forward to connecting and collaborating at the 2025 AAD Congress as we work together to transform dermatological care.

If you are a healthcare professional attending AAD, join us at our scientific symposia or visit our booth to learn more. We look forward to meeting you in Orlando!

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��ֳ�Cares® - a decade of support and beyond

Vandenbruaene Nathalie — Tue, 11 Feb 2025 16:20:36 +0000

��ֳ�Cares® - a decade of support and beyond

Sarah Mertens, ��ֳ�Cares®

Vandenbruaene … Tue 11/02/2025 - 17:20

��ֳ�Cares® proudly celebrates its 10th anniversary - a decade dedicated to supporting people living with severe diseases, caregivers, and healthcare providers with compassion and professionalism.

��ֳ�Cares® is ��ֳ�'s global support center, functioning as a critical bridge between our company, healthcare providers, and the people we serve. Established in 37 countries, ��ֳ�Cares® resolves over 58,000 inquiries annually, providing real-time, localized assistance around ��ֳ�'s medicines and solutions. The inquiries handled by ��ֳ�Cares® span diverse areas, including supply, medical, customer service, safety and product quality concerns.

��ֳ�Cares® interacts with a variety of our stakeholders, including physicians and patients who make up 25% of interactions. This customer-centric approach helps ��ֳ�Cares® foster a positive impact on people’s lives as they often reach out with specific questions about their medication. Underpinning ��ֳ�'s commitment to patients, ��ֳ�Cares® strives to provide tailored information, materials, services, or support. This approach results in 79% of people accepting additional resources, showcasing the impact of these personalized measures.

Most inquiries are resolved the same day, offering a one-stop solution for people’s needs via immediate and accurate information about ��ֳ� medications. This swift response rate ensures stakeholder satisfaction, with 96% of people calling us rating ��ֳ�Cares® +9 out of 10.

��ֳ�Cares® also transforms everyday interactions into valuable insights, using AI tools to pinpoint trends and identify actionable insights that can shape our company’s priorities. For example, the service has identified an opportunity to better support pharmacists with dedicated resources, helping to improve access to our medicines and address misconceptions about medicine use and safety through educative material and campaigns.

In essence, ��ֳ�Cares® is more than a support center. It's a service that reinforces our engagement towards people living with severe diseases and healthcare providers, exemplifying ��ֳ�’s commitment to improving lives through efficient service and insightful data utilization.

This milestone is a testament to ��ֳ�’s unwavering commitment to improving lives, and it serves as a reminder of the incredible impact that consistent and empathetic support can have on communities worldwide.

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��ֳ�Cares support center helpline

Comment:

Posted by Peter Arthur, 19 February 2025

Proud of having been part of the ��ֳ�Cares family, keep up the good work you are doing every day
🤗👏👏👏

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More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies

Andrea_Puletto — Mon, 10 Feb 2025 09:50:16 +0000

More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies

Andrea Wilkinson, Global Epilepsy & Rare Syndromes

Andrea_Puletto Mon 10/02/2025 - 10:50

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 CDKL5 deficiency 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.

With International Epilepsy Day on 10 February 2025, followed closely by Rare Disease Day on 28 February, themed ‘More than you can imagine’, it’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.

Looking beyond seizures

Dravet syndrome (DS), Lennox-Gastaut syndrome (LGS) and CDKL5 deficiency disorder (CDD) 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.

These DEEs don’t end with the individual — 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.

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.

Although people’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.

Working together to address the non-seizure burden

At ��ֳ�, 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, enhance 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.

‘More Than You Can Imagine’ – A theme to remember

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 “epilepsy”, their complexities extend far beyond seizures. Recognizing these challenges – and addressing them – could change what is often an overwhelming diagnosis into a life supported by compassionate resources and initiatives.

We invite you to help us increase understanding of the broader impacts of epilepsy. Whether it’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, you’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.

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neurology epilepsy TK2d

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��ֳ�’s commitment to understanding Sudden Unexpected Death in Epilepsy

Vandenbruaene Nathalie — Wed, 16 Oct 2024 10:42:44 +0000

��ֳ�’s commitment to understanding Sudden Unexpected Death in Epilepsy

Andrea Wilkinson, Patient Engagement, Epilepsy & Rare Syndromes

Vandenbruaene … Wed 16/10/2024 - 12:42

Sudden Unexpected Death in Epilepsy (SUDEP) occurs when a person living with epilepsy dies suddenly and prematurely for no known obvious reason. It is a leading cause of death in people with epilepsy and represents an unimaginable outcome for anyone living with epilepsy and their loved ones. Discussing SUDEP is challenging, yet addressing this tough subject reflects the expressed needs of individuals affected by epilepsy.

SUDEP Action Day on October 16 is dedicated to raising awareness of the risk of sudden death in epilepsy and aspires to stop preventable deaths. A time when epilepsy organisations across the world come together with a singular purpose: to encourage all healthcare providers, individuals with epilepsy and their families to actively engage in conversations about minimizing SUDEP risks and participating in shared decision-making. The benefits of shared decision-making include improved decision quality, empowering patients to make more informed choices and better treatment consistency.

For ��ֳ�, SUDEP Action Day is aligned with our mission to advance understanding around epilepsy, improve awareness, and elevate the unmet needs of patients and their families. Under this year’s theme of #OurRisksOurRights, we support the goal that every person living with epilepsy should know their SUDEP risk. Integral to this is early epilepsy diagnosis, access to plain language information, medication adherence, updated emergency seizure action plans and the need for improved care coordination.

Understanding SUDEP risk
While no one knows exactly what causes SUDEP, researchers are investigating several possibilities, like the effect of seizures on the heart, the brain and breathing. Certain factors can increase the likelihood of SUDEP, such as frequency of generalized tonic-clonic seizure (GTCS), nocturnal seizures, medication adherence and/or failure to adequately control treatment-resistant seizures.

According to the International League Against Epilepsy, optimizing seizure control, especially GTCS, is the most important measure in reducing a person’s risk of SUDEP.5 However, despite currently available antiseizure medications, 1 in 150 people with uncontrolled seizures die from SUDEP every year. Some people with rare and complex epilepsies continue to experience frequent and uncontrolled seizures and remain at a higher risk of SUDEP.

For example, children with Dravet syndrome are 15 times more likely to die suddenly. Those with Lennox-Gastaut syndrome have a 24 times greater likelihood of premature death compared to individuals with other childhood epilepsies. Emerging research estimates that SUDEP rate is 2.8 per 1,000 person-years.

It is equally shocking to learn that the risk of early death and SUDEP is higher among lower income groups. In fact, people with epilepsy who are in poor health or live on a low income tend to live about 17 years less than average and this is especially true for young and middle-aged adults.

��ֳ�’s commitment to SUDEP
At ��ֳ�, we are aligned with the patient and healthcare community’s desire to reduce preventable deaths from epilepsy by investing in research partnerships, new technologies and championing disease awareness.

SUDEP occurs most often at night or during sleep, leaving many unanswered research questions. ��ֳ� supports Neurava, a medical device start-up that has developed a first-of-its-kind device work around the neck and bicep at night which has the potential to save lives by identifying key biological signals related to epilepsy, tracking seizures and impending SUDEP risk.

Global clinical guidelines recommend counseling people with epilepsy (PWE) on sudden unexpected death in epilepsy (SUDEP). ��ֳ� proudly collaborated with the Child Neurology Foundation alongside Partners Against Mortality in Epilepsy and 20 other organizations to develop the Clinical Toolkit for Preventing Epilepsy Deaths. The toolkit focuses on behavior change by offering health providers and patients tips for on how to talk about mortality risk factors, preventative measures, and treatment options.

Looking ahead
As we advance our understanding of human biology and neurological diseases, we aim to drive innovation in epilepsy alongside clinicians, academics and patient communities. By specifically targeting the mechanisms underpinning SUDEP, we hope to identify the causes and potential biomarkers of SUDEP and reduce the major risk factors for this devastating outcome.

On SUDEP Action Day, we stand with the community and encourage everybody to raise awareness of the risks of sudden death in epilepsy. 

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Sudden Unexpected Death in Epilepsy SUDEP epilepsy

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Redefining immunology care: our pledge to patients and the healthcare community

Vandenbruaene Nathalie — Mon, 23 Sep 2024 08:15:38 +0000

Redefining immunology care: our pledge to patients and the healthcare community

Camille Lee, U.S. Immunology

Vandenbruaene … Mon 23/09/2024 - 10:15

Throughout my career and now in my role as head of U.S. Immunology at ��ֳ�, the stories I hear from patients inspire my work each day. At ��ֳ�, we are dedicated to developing solutions for people living with chronic immune-mediated diseases, including psoriatic arthritis (PsA)—which can affect about 30 percent of people living with psoriasis—as well as lesser-known forms of arthritis like non-radiographic axial spondyloarthritis (nr-axSpA) and ankylosing spondylitis (AS).

At the heart of our commitment to addressing gaps in care lies innovation. ��ֳ� is proud to be advancing care with a new solution now available for healthcare providers and their patients.

We know severe rheumatic diseases not only affect physical health but can also impact a person’s ability to take part in everyday activities such as participating in hobbies and recreational activities, socializing with loved ones, and completing household chores. People living with rheumatic diseases often struggle to find long-term disease management solutions that work for them.

That’s why our focus in Immunology at ��ֳ� is developing and providing treatments so that people living with rheumatic or dermatologic diseases, particularly those who have struggled with treatment options, can strive to live their best lives, tackling the activities that once felt like a burden.

��ֳ� is also leading the way in setting higher standards for treatments across immunology. In our PsA clinical trials, we used the American College of Rheumatology (ACR)50 endpoint, which signifies a 50% improvement in symptoms and disease, as a measure of success. This endpoint is more rigorous than the more commonly used ACR20, the 20% improvement marker. By setting this higher standard, we prioritize developing medicines that make a significant impact on patient health.

As our pipeline grows, our approach will continue to evolve to meet the needs of the patient communities we aim to serve. I am confident our bold and exciting research programs will help change the future of immunology care within the next decade.

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rheumatic diseases Immunology psoriatic arthritis non-radiographic axial spondyloarthritis ankylosing spondylitis

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Shaping a better future for women living with epilepsy

Vandenbruaene Nathalie — Wed, 03 Jul 2024 13:56:35 +0000

Shaping a better future for women living with epilepsy

Andrea Wilkinson, Epilepsy & Rare Syndromes Patient Engagement

Vandenbruaene … Wed 03/07/2024 - 15:56

Throughout life, women face numerous societal challenges that accompany the physiological changes that manifest into a unique life story. These physiological transformations range from puberty to menopause and beyond. For many women, the childbearing years are the most profound including contraception, pregnancy, childbirth, and post-pregnancy. Each phase carries opportunities and challenges, and for women living with a chronic disease like epilepsy, this journey can be complex and intimidating.

Lack of information

Women living with epilepsy often navigate the motherhood maze with inadequate information on critical aspects about their anti-seizure medications (ASMs) in birth control discussions, fertility planning, pregnancy, breastfeeding, postpartum and outcomes in the child after in utero exposure to ASMs. This lack of knowledge can cast a shadow of uncertainty over their reproductive journey, forcing too many women to make the difficult choice between managing their epilepsy and planning for a family, with some women deciding against having a family due to the challenges.

We are proactive in empowering women with epilepsy to advocate for themselves in discussions about seizure treatment and family planning. We share plain language summaries of our clinical trials’ results, publish other pregnancy-related research and co-create discussion guides that equip women and healthcare professionals to ask the right questions, at the right time.

Advancing the science

At ��ֳ�, we strongly believe in the importance of understanding and addressing the unmet needs of women during their reproductive journey. We are driven by the idea of doing more to support them during these pivotal years.

��ֳ� was among the first companies to address the lack of data and research for women. Our commitment to understanding and addressing the unique challenges that women living with epilepsy face has inspired us to strive to advance the science, specifically targeting their reproductive years.

Our aim is clear: to empower through information that enables women to make informed decisions about their reproductive and post-partum health care and beyond, and to know the potential impact those decisions could have on their child. We conducted a deep dive into our global safety database to assess the risk of our medications to the fetus as well as pharmacokinetic studies of our anti-seizure medications to check plasma and breast milk concentration during and after pregnancy.

Collaborating for change

For many women with epilepsy, the inability to access pertinent information can lead to a feeling of isolation. To combat this, we have turned our focus towards building connections and sharing knowledge. In our quest to bring about meaningful change, we collaborate with strategic partners and stakeholders on various projects, including:

ConcepTION, a project funded by the Innovative Medicines Initiative (IMI) aimed at reducing uncertainty about the effects of medication used during pregnancy and breastfeeding.
Coalition to Advance Maternal Therapeutics (CAMT), a project spearheaded by the Society for Maternal Fetal Medicine focusing research on pregnancy and lactation.
Feedback on the FDA’s pregnancy safety study framework.
BRIDGE (Better Research, Information and Data Generation for Empowerment), a multidisciplinary group dedicated to closing the information gap necessary for women living with chronic diseases to receive optimal care.
Collaborating with the International Bureau of Epilepsy’s Women and Epilepsy Working Group to bring more information to women living with epilepsy.

Through in-depth research, strategic collaborations, and building awareness, ��ֳ� is laying the foundation for a future where every woman living with chronic disease feels empowered to make informed decisions about her healthcare.

For more information about epilepsy, visit Epilepsy & Rare Syndromes at ��ֳ� | ��ֳ�

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epilepsy family planning seizures

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Turning awareness into action for Myasthenia Gravis Awareness Month

Vandenbruaene Nathalie — Wed, 05 Jun 2024 13:28:50 +0000

Turning awareness into action for Myasthenia Gravis Awareness Month

Manuela Maronati, Rare Diseases Europe

Vandenbruaene … Wed 05/06/2024 - 15:28

Every day, over 700,000 people across the globe live with generalized myasthenia gravis (gMG), a rare disease that most people have never even heard of. gMG occurs when the body’s immune system mistakenly attacks the connections between nerves and muscles. It causes weakness in the muscles that control some of our most basic movements, like seeing, walking, talking, breathing and even smiling. The multitude of symptoms contribute to the emotional toll and daily struggles that people living with this disease face.

Despite this, like many rare diseases, there is a lack of wider knowledge around gMG. This is why it’s especially important that we join forces with the gMG community to recognize MG Awareness Month this June and support the movement to “Turn Awareness into Action.” At ��ֳ�, we are committed to driving real change for people living with gMG.

Prioritizing collaboration with patients
In pushing for progress in gMG, early collaboration with the patient community is essential. This collaboration can take many forms – from working with patients to shape clinical trials, to fostering compassionate care and integrating the patient voice in research. Patients are the experts in living with their condition – by drawing on their lived experiences we can ensure that the patient voice is at the heart of our work to help improve treatment and care for those with gMG.

An example of ��ֳ�’s collaboration with the community is our partnership with expert patients to bring the lived experience of gMG to the attention of the medical community with peer-reviewed publications to highlight the real-life experiences of people living with gMG and strengthen the bond between the scientific and MG patient community. In gMG, the patient population is small and knowledge on the disease is limited, meaning amplifying the patient voice is essential to improving outcomes for those living with this often debilitating disease.

Patient quality of life can often be underestimated
gMG is a complex disease with a range of symptoms – as a result, several aspects that impact patient quality of life can often be underestimated, including fatigue, emotional impact, and difficulties in swallowing or speaking. ��ֳ� sought to address this through collaboration with the patient community which culminated in the development of the MG Symptoms PRO tool, which provides a quantifiable means of assessing MG symptoms that are most relevant to patients, which are not included in other PRO measures like physical fatigue, aligning with patients' lived experiences and paving the way for the development of enhanced management strategies.

Innovating to bring MGS-PRO tool to life has been crucial in helping address these issues. By approaching these challenges from a holistic, patient-centered lens, we’ve been able to assess many symptoms that matter to people living with gMG and help understand the impact of gMG on their daily lives.

However, transformational change requires more than innovation alone. We see meaningful progress when there is shared purpose, a unified approach, and ongoing collaboration and dialogue between stakeholders.

At ��ֳ�, we’re focused on reshaping the MG landscape, and recognize that collaboration with the MG community can accelerate that mission. Through listening, learning, and engaging, we’re committed to accelerating the development and access to life-changing treatments for people living with MG, turning awareness into action. Join us in making a difference by getting involved in MG Awareness Month!

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MG Awareness Month generalized myasthenia gravis Myasthenia Gravis gMG

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Advancing neurodegenerative disease research at AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders

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Our Commitment to Advancing Treatments for TK2d

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Exploring the future of immuno-dermatology care: ��ֳ� at AAD Annual Meeting 2025

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��ֳ�Cares® - a decade of support and beyond

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‘More Than You Can Imagine’ – A theme to remember

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��ֳ�’s commitment to understanding Sudden Unexpected Death in Epilepsy

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Redefining immunology care: our pledge to patients and the healthcare community

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Shaping a better future for women living with epilepsy

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Advancing neurodegenerative disease research at AD/PD™ 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders

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Innovating in neurology: ������ֳ�'s commitment shines at AAN 2025

A focus on valuable solutions

Showcasing innovation at AAN

Our commitment to science and care

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Our Commitment to Advancing Treatments for TK2d

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Exploring the future of immuno-dermatology care: ������ֳ� at AAD Annual Meeting 2025

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������ֳ�Cares® - a decade of support and beyond

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More Than You Can Imagine: Shedding Light on the Burden of Rare Epilepsies

Looking beyond seizures

Working together to address the non-seizure burden

‘More Than You Can Imagine’ – A theme to remember

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������ֳ�’s commitment to understanding Sudden Unexpected Death in Epilepsy

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Redefining immunology care: our pledge to patients and the healthcare community

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Shaping a better future for women living with epilepsy

Lack of information

Advancing the science

Collaborating for change

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Turning awareness into action for Myasthenia Gravis Awareness Month

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Innovating in neurology: ��ֳ�'s commitment shines at AAN 2025

Exploring the future of immuno-dermatology care: ��ֳ� at AAD Annual Meeting 2025

��ֳ�Cares® - a decade of support and beyond

��ֳ�’s commitment to understanding Sudden Unexpected Death in Epilepsy