Muscular Disorders Drug Development Summit
Muscular Disorders Drug Development Summit | January 26-28, 2027 | Boston, MA
The muscular disease field is entering a pivotal new chapter. Following a year of significant debate around Sarepta’s Elevidys, the industry is rapidly evolving as a new wave of gene therapies, RNA medicines, stem cell therapies, and small molecules advance through development. With anticipated regulatory milestones from companies including Regenxbio, Capricor, and Avidity Biosciences, alongside growing investment and renewed pharmaceutical interest, momentum has never been stronger across muscular dystrophies, Pompe disease, and related myopathies.
The inaugural Muscular Disorders Drug Development Summit is the only industry-focused forum dedicated to helping biopharma leaders accelerate the translation, clinical development, and delivery of next-generation therapies for muscle disorders.
Be the first to access the newly launched event program.
Across three days of expert-led presentations, interactive discussion, and dedicated networking, attendees will:
- Discover lessons learned from approved and late-stage muscular disease therapies
- Explore emerging modalities beyond traditional gene therapy
- Advance biomarker strategies and functional outcome assessments
- Innovate targeted delivery approaches to improve efficacy and reduce off-target effects
- Leverage real-world data to identify meaningful, patient-relevant endpoints
- Build partnerships that can accelerate clinical and commercial success
Join 60+ industry experts and leading organizations including Servier, Dyne Therapeutics, Sanofi, Biogen, and Avidity Biosciences for three days of collaboration focused on overcoming the biggest challenges in muscular disorder drug development.
Whether you’re advancing therapies for DMD, FSHD, DM, LGMD, Pompe disease, or other rare muscle disorders, this is the must-attend meeting to benchmark strategies, forge new partnerships, and help bring transformative treatments to patients faster.