[Updated 8/13/2020, 3:19 p.m.] Duchenne muscular dystrophy (DMD) patients whose disease is characterized by a particular genetic mutation now have a new treatment option following the FDA’s Wednesday approval of a drug developed by NS Pharma.

DMD is a genetic disorder that leaves patients unable to produce dystrophin, a protein key to muscle function. NS Pharma designed its drug, an antisense oligonucleotide called viltolarsen (Viltepso), to get a cell’s protein-making machinery to skip over the mutation so it still can produce dystrophin, albeit much less than those without any such mutations typically produce.

This rare mutation, which occurs in… Read more »

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