The Neuromuscular Drug Development Summit (NMD) covers topics such as:
- Potential therapies for orphan neuromuscular diseases - mechanisms of action and pre-clinical studies
- The translation of Gene therapy in phase I/II studies - dosing, safety and pre-clinical efficacy
- The scientific rationale for choosing appropriate muscle biomarkers
- Clinical trial recruitment hurdles and issues in clinical meaningful endpoints
- The role of inflammation in neuromuscular disease
The Neuromuscular Drug Development Summit (NMD) might be held in Boston, MA, United States in 2021.