Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Research highlights that the DMD gene is the largest known human gene, featuring 79 exons (coding regions). The gene’s large size means it is prone to alterations that may result in DMD.

Avidity is hoping to follow in the footsteps of Sarepta, which has launched a series of exon-skipping therapies for DMD, targeting exons 51, 53, and 45, that brought in around $1 billion in sales ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...