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 ...
NS Pharma’s Viltepso (viltolarsen) has been approved in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The DMD gene is made up of 79 exons ...
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 ...