Musculoskeletal

The Gene Therapy for Muscular Disorders Summit took place in April this year, and is the field’s only industry-focused event specifically designed to help gene therapy developers successfully target the muscle. With a disease-agnostic agenda, but highlighting novel research in Duchenne’s (DMD), Limb-Girdle (LGMD), Pompe Disease and X-Linked Myotubular Myopathy (XLMTM), the expert industry speakers from the likes of Solid Biosciences, Sarepta Therapeutics, Astellas Gene Therapies and Pfizer spent 4-days discussing the fields hottest topics from navigating toxicity to state-of-the-art muscular targeting.