FAQs

Yes. The Muscular Disorders Drug Development Summit is uniquely focused on the scientific, translational and clinical challenges involved in developing therapies for muscular disorders. Unlike broader rare disease conferences, this event is dedicated to advancing treatments for indications including DMD, FSHD, DM1, LGMD, Pompe disease and related myopathies.

Yes. The Muscular Disorders Drug Development Summit explores cross-modality innovation, including gene therapies, RNA therapies, stem cell approaches, small molecules and other emerging treatment platforms shaping the future of muscular disorder drug development.

Yes. The agenda for the Muscular Disorders Drug Development Summit spans a broad range of muscular disorders, including Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy (DM1), limb-girdle muscular dystrophy (LGMD), Pompe disease and additional muscle-related conditions.

Yes. The Muscular Disorders Drug Development Summit features dedicated workshops designed to help attendees tackle key translational and clinical development challenges, including biomarker strategies, targeted muscle delivery, clinical trial design and endpoint selection.

Yes. Networking is a core part of the Muscular Disorders Drug Development Summit. Attendees can benefit from more than eight hours of dedicated networking with experts spanning biotech, pharma, translational research, clinical development and enabling technology providers, through dedicated panel discussions, poster sessions and networking breaks.

Yes. The Muscular Disorders Drug Development Summit brings together senior leaders and scientific experts from organizations including Sarepta Therapeutics, Servier, Sanofi, Biogen, Dyne Therapeutics, Avidity Biosciences and others working at the forefront of muscular disorder drug development.

Yes. The Muscular Disorders Drug Development Summit includes a dedicated scientific poster session where researchers can showcase novel findings, engage with industry peers and generate valuable scientific discussions around muscular disorder drug development.

The latest agenda, speaker line-up and event information for the Muscular Disorders Drug Development Summit can be downloaded directly from the event website, allowing attendees to explore the most up-to-date program content and session details.

See the Full Event Guide here.