Earlier this year, NS Pharma received clearance from the U.S. Food and Drug Administration (FDA) for their Phase II study for an exon 44 skipping candidate for treating Duchenne muscular dystrophy.

Recently they announced that they have also received clearance to initiate a Phase I/II study for NS-050/NCNP-03, their exon 50 skipping candidate, assessing dystrophin production, muscle strength, mobility and functional exercise capacity.

“Exon skipping therapies have the potential to treat a wide range of patients with Duchenne, but more than half of Duchenne patients potentially amenable to exon skipping therapy have no approved treatment options that target their specific mutation.

That is why it is exciting that this year we are beginning studies of two treatments that have the potential to reach even more Duchenne patients with targeted exon skipping therapies.”

Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago

Details about study enrollment for NS-050/NCNP-03 in the US will be shared in the second half of 2023, and Defeat Duchenne Canada will continue to work with NS Pharma for Canadian inclusion.