Duchenne Muscular

Stealth BioTherapeutics Corp (NASDAQ: MITO) presented new data demonstrating the potential synergistic relationship between elamipretide and an exon-skipping phosphorodiamidate morpholino oligomer (PMO) in the X-linked muscular dystrophy (mdx) animal model.  The data were...

The FDA has granted Orphan Drug Designation to RegenXbio Inc's (NASDAQ: RGNX) RGX-202, potential one-time gene therapy for Duchenne muscular dystrophy (Duchenne).  Duchenne is a genetic disorder characterized by progressive muscle degeneration and weakness due to the...

Solid Biosciences Inc (NASDAQ: SLDB) shares were trading close to their historical lows Thursday following the release of data from a Phase 1/2 dose-escalating trial of its Duchenne muscular dystrophy candidate SGT-001. DMD, caused by the absence of a protein called dystrophin that aids in...