What Can You Expect at the Inaugural Muscular Disorders Drug Development Summit?
Driving the Next Generation of Therapies for Patients with Muscular Disorders
The Muscular Disorders Drug Development Summit brings together leading experts from biopharma, academia, and investment organizations to tackle the most pressing challenges in development for myopathy therapeutics. Explore the latest advances in biomarker and endpoint selection, translational research, and emerging modalities such as exon skippers and small molecules.
Through expert-led presentations, real-world case studies and collaborative discussions, gain practical insights and shape your strategies to overcome scientific, clinical and regulatory barriers. Leave with new connections, actionable answers, and a clearer path to accelerating transformative therapies for patients living with muscular disorders.
What to Expect?
Explore Emerging Modalities for Muscular Disorders R&D
Evaluate the benefits and drawbacks of small molecules, gene therapies and RNA-targeting therapeutics. Consider the future of combination therapies with Edgewise, and understand how different modalities can affect immunogenicity, biodistribution and efficacy with Bonito Biosciences.
Shape your Clinical Trial Design with Tested Expert Strategies
Learn how Sarepta navigates patient heterogeneity in rare disease trials for DM1 and FSHD, and how Avidity begin trial design considerations in the preclinical phase with exon skippers for DMD, to increase the likelihood of clinical success.
Learn to Think Like an Investor & Derisk Drug Development
Understand the investor mindset with insights from venture philanthropic organization, Solve FSHD, to build confidence in your drug development program, whilst developing stronger investor relationships.
Investigate Novel Biomarkers & Endpoint Selection Tools
Discover emerging biomarkers uncovered by Dyne Therapeutics to innovate the delivery of RNA therapeutics and enhance their real-world impact for patients.
Enhance Specificity & Biodistribution in Muscle Delivery
See how NuCyRNA Therapeutics is engineering novel targeting technologies to enhance payload delivery for DM1 to overcome challenges with long-term accumulation and toxicity.
Discover the Full Scientific Program:
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.
Pre-Conference Workshop Day
Join experts from leading biotech and pharma organizations already attending the workshop day, including Sanofi, PTC, Edgewise, Lundbeck & UCB to tackle shared translational and clinical development challenges across muscular disorders. Secure valuable networking opportunities ahead of the main conference and gain practical insights that can strengthen your development strategy.
Get ahead of the networking and build meaningful relationships before the main conference begins
Engage directly with peers tackling muscular dystrophy, Pompe disease and related myopathy development challenges
Collaboratively discuss clinical trial design, biomarker strategy, endpoint selection and translational hurdles
Exchange lessons learned across gene therapy, RNA therapeutics, stem cell approaches and small molecules
Leave with actionable insights from content not available within the main conference agenda, to accelerate your own development programs
Maximize your three-day experience by establishing valuable connections early
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
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.
Partner With Us
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.