Going beyond genetic medicines for muscular dystrophy

Muscular dystrophies are poised for what could turn out to be a step change in the field, even if only a fraction of the rich pipeline in the clinic makes it over the finish line. The twist is that the major impact might come from compounds addressing the disease biology as opposed to its genetic roots.

Across the class of muscular dystrophies, at least 20 programs covering 18 targets aim to treat the diseases by addressing muscle cell damage or its downstream consequences, rather than the genetic etiologies of the diseases. Of these, at least 13 are small molecules. The remaining programs, including those based on causative mutations, span at least seven modalities including antibodies, oligonucleotides and stem cell-derived therapies...