How venture philanthropy is driving modality innovation for DMD

The DMD market could be nearing an inflection point as new therapies enter the clinic based on two technologies that promise to overcome the challenges of first-generation products. Moreover, the innovations behind the new treatments should have effects that extend well beyond the indication.

One therapeutic modality — exon-skipping antisense oligonucleotides — has already led to four approved therapies for Duchenne muscular dystrophy, and a second modality, gene therapy, is nearing the market...