Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. The meeting offers a dynamic platform for sharing new data, fostering collaborations, and advancing the science behind therapies for diseases like Duchenne and Becker muscular dystrophy. This year’s event in Vienna, Austria, features talks focusing on neuromuscular diseases as multisystemic disorders, multidisciplinary disease management, and advances in therapies and drug development.

Below are highlights from the 2025 meeting, updated daily with information from members of PPMD’s team attending the meeting in Vienna:

Psychological Care in Neuromuscular Diseases

This session highlighted the vital role of psychological care in managing neuromuscular diseases like Duchenne and Becker muscular dystrophy. Psychologist Sabrina Sayah discussed key emotional moments along the patient and family journey—from diagnosis and changes in mobility to major life transitions and treatment decisions—while also addressing challenges unique to rare, progressive, and genetic conditions, such as uncertainty, feeling unseen, and navigating family impacts. She emphasized that psychological support should be personalized and integrated into the full care plan, helping individuals and families find meaning, balance, and connection. For families, this approach is especially important as new therapies expand possibilities, ensuring that emotional well-being remains a core part of care alongside physical health.

Advances in Therapies and Drug Development

Improving AAV Vectors for Neuromuscular Disease Indications

In this session, Dr. Melissa Spencer provided an overview of the current landscape of adeno-associated virus (AAV) vectors, the delivery systems used in many gene therapies for neuromuscular diseases. She discussed how researchers are working to optimize AAVs to more effectively deliver therapeutic genes into muscle while also reducing the immune risks that can come with viral delivery. Dr. Spencer highlighted both the promise and the ongoing challenges of AAV-based therapies, including issues related to dosing, efficacy, and safety, and shared recent research aimed at improving vector design and delivery strategies to make gene therapies more effective and accessible. Her presentation underscored the importance of continued innovation to refine AAV technology—work that holds great potential for advancing safer, more durable treatments for individuals living with Duchenne, Becker, and other neuromuscular conditions.

Leveraging the Muscle Fusogens for Gene Delivery

Friday morning’s sessions at WMS highlighted exciting progress in the delivery of dystrophin restoration therapies. Dr. Douglas Millay shared new pre-clinical research from his lab exploring how to harness natural muscle biology to improve how treatments reach and repair muscle tissue. His team is investigating the potential use of lentivirus and non-viral vectors to deliver micro-dystrophin transgenes—an approach that could one day offer alternative or complementary options to current gene therapy strategies. While these studies are still in early stages, they reflect the ongoing innovation and determination within the research community to develop more effective, targeted therapies for individuals living with Duchenne and Becker muscular dystrophy.

PPMD Posters

TREAT-NMD Neuromuscular Network: Variations in DMD Mutation Distribution in the Duchenne Muscular Dystrophy Dataset

The Duchenne Registry is a member of the TREAT-NMD international neuromuscular registry network, which means that data provided by individuals living with Duchenne and Becker and their families consistently supports research on a global stage.  The Duchenne Subgroup of TREAT-NMD joined together to study the frequency and distribution of different types of DMD gene variants around the world. This large-scale, international dataset highlights significant regional variation in DMD variant types, with direct implications for therapy eligibility and clinical trial planning. Just like all other uses of this data, information provided for TREAT-NMD research projects is de-identified. To all Registry participants, thank you for sharing your data and making this research possible!

Care of Dystrophinopathy Carriers: Survey Results of Provider Practices in the United States

Scavina, M., Banks, K., Cappa, R., Chambers, C.

A Qualtrics survey was distributed to members of the National Society of Genetic Counselors, Parent Project Muscular Dystrophy Certified Duchenne Care Center (CDCC) Network, and American Academy of Neurology to evaluate current care of dystrophinopathy carriers throughout the country.  Questions ranged from age of initial genetic testing, care practices and recommendations, and preferred terminology. Additionally, the survey found that most providers did not screen for learning differences and mental health concerns, indicating an area of clinical care requiring awareness and attention.

The Spirit of Collaboration at WMS

Reflections from PPMD’s Founding President Pat Furlong

Thirty years ago, Professor Victor Dubowitz helped bring to life the first World Muscle Society (WMS) meeting. His vision was simple yet transformative: to unite researchers and clinicians — “mouse doctors” and “people doctors” — so they could learn from one another, share discoveries, and work together for the benefit of patients. Dr. Dubowitz was more than a brilliant physician and scientist; he was a tireless advocate for individuals and families living with neuromuscular disorders. An early adopter of steroid therapy in Duchenne muscular dystrophy, his team explored intermittent dosing schedules — including the common “10 days on, 10 days off” schedule — to balance benefit while reducing side effects. These studies reflected his lifelong commitment to thoughtful, patient-centered care and his constant pursuit of safer, more effective treatments.

At his core, Dr. Dubowitz was a pioneer who believed that progress in the lab meant little unless it improved life at the bedside. He built bridges between disciplines, convinced that collaboration and shared learning were essential to move the field forward. The WMS meeting was born from that conviction — and remains a living legacy of his passion, curiosity, and compassion. We at PPMD have had the honor of hosting Dr. Dubowitz as a speaker at our Annual Conferences, underscoring his lifelong commitment to engaging directly with patients, families, and advocates. As Duchenne research has advanced and new partners from biopharma, academia, and advocacy have joined the mission, WMS has continued to grow inclusively — a reflection of Dr. Dubowitz’s belief that real progress depends on collaboration, and that patients and families must remain at the heart of this work.

Today, PPMD continues to attend WMS in that same spirit: to learn, to share, and to collaborate. Bringing the patient advocacy voice into scientific and clinical conversations — and especially into a space like WMS — changes everything. It reminds every person in the room why this work matters: that behind every data point is an individual, a parent, a family hoping for more time, more strength, more life. Advocates bring urgency, context, and perspective that science alone cannot supply. We help ensure that the research being pursued reflects what truly matters to families — meaningful outcomes, improved quality of life, and access to care.

When advocacy is part of the discussion, priorities shift from theoretical success to practical, human impact. These conversations influence how clinical trials are designed, how regulators and investors think about therapies, and how care evolves in real time for families around the world. They remind the scientific community that communication and compassion are not secondary to discovery — they are essential to it. When advocates and experts sit at the same table — because it is our table, after all — science moves faster, care improves, and families feel seen and heard. That is the true value of patient advocacy: bringing the patient voice into the heart of medicine, and carrying forward Dr. Dubowitz’s enduring belief that by working together, we can — and will — end Duchenne.