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

The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.

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a treatment for Duchenne muscular dystrophy or DMD in patients with exon 44 skipping amenable mutations. The company announced it has received authorization from the United Kingdom's Medicines and ...