Strengthening Translational & Clinical Development of a New Wave of Genetic, Stem Cell, Small Molecule & RNA-Based Muscle Disease Therapies
After a year marked by uncertainty around Sarepta's Elevidys, the field is on the verge of new era shaped by cross-modality innovation and meaningful progress, with FDA submissions anticipated from Regenxbio, Capricor and Avidity that could transform therapeutic options for muscular dystrophies.
With investments including Servier’s $2.65 billion buyout of Edgewise signposting reignited pharma interest and funding opportunities, muscle disease drug developers are poised to double down on investments across their preclinical, translational and clinical programs in 2026 and beyond.
Take advantage of this timely opportunity to join 60+ industry experts spanning muscle disease biology, translation, clinical development and more at the Muscular Disorders Drug Development Summit for three days of focused collaboration designed to:
- Explore emerging modalities beyond gene therapy
- Discover lessons learnt from developers of approved drugs
- Enhance biomarker measurements of muscular function and activity
- Innovate more targeted delivery to specific muscles while avoiding off-target tissues
- Leverage real-world data to uncover patient-relevant endpoints
With momentum building, don’t miss your chance to learn from Servier, Dyne Therapeutics, Sanofi, Biogen, Avidity Biosciences and more at the only forum dedicated to addressing biopharma’s most pressing challenges in advancing novel therapies for muscular dystrophies, Pompe disease and related myopathies.
Hear From Your Industry Peers:
I’m looking forward to listening, learning and sharing to better serve the muscular dystrophy communities.
Nitza Thomasson, Executive Director, Global Head of Research & Development, Servier
Muscular Disorders Drug Development Summit Expert Speaker
High quality of talks and excellent focus on networking. Good size for discussion and making new connections.
Lundbeck, 7th CNS Drug Delivery Summit Past Attendee
Very open environment for collaboration and communication. First time I’ve been to an industry focused event and I was positively impressed.
Athira Pharma, 5th Oligonucleotides for CNS Summit Past Attendee
This meeting exceeded my expectations. I benefited greatly from both the presentations and the networking opportunities, gaining valuable scientific knowledge and professional insights.
Stoke Therapeutics, 5th Oligonucleotides for CNS Summit Past Attendee
Attending Companies Include
Explore the Full Event Guide
- Explore the evolving cross-modality muscular disorder therapeutic landscape
- Shape your clinical trial strategies with peer-led success stories
- Learn how to derisk your drug development and build translational confidence
- Investigate innovative endpoint selection tools and novel biomarkers
- Discover expert technologies to enhance biodistribution and muscle delivery
Official Partners
Covering the Full Spectrum of Muscular Disorders Drug Development
From Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD) to myotonic dystrophy (DM1), limb-girdle muscular dystrophy (LGMD), Pompe disease, and related myopathies, the Muscular Disorders Drug Development Summit brings together leaders driving innovation across the muscular disease landscape. Explore shared development challenges, indication-specific insights, and opportunities to accelerate therapeutic advancement across multiple disease areas.
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Explore the Agenda
Discover exactly how industry leaders are facing the most important development challenges – from targeted muscle delivery, patient pool selection and preclinical model translatability.
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Position your solutions in front of decision-makers actively looking to optimize their muscular disorder drug development, to become a trusted partner and build lasting relationships to support long term success.
Join Biopharma Leaders
Connect with the Muscular Disorder community and be part of the hottest conversations with like-minded attendees and gain access to insights only shared in our innovative face-to face networking sessions.