The Japanese pharma had one asset rejected by the FDA and withdrew a regulatory application for another, but already this ...

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

Achondroplasia, which affects around one in 20,000 babies, has just one approved treatment: BioMarin’s Voxzogo. However, new investigational treatments are vying to compete in the area.

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