Once a Wild Idea, Successful First-Generation Exon-Skipping Therapies Pave the Way for Personalized Treatments

In 1996, MDA provided a small starter grant to Dr. Steve Wilton, a primary investigator at the University of Western Australia in Perth, for research into what scientists then thought was a radical process to address the effects of certain gene mutations. A quarter of a century later, this process — exon skipping — has . . .

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FDA Approves NS Pharma’s Viltepso for Treatment of DMD Amenable to Exon 53 Skipping

Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .

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NS Pharma Launches Expanded Access Program for Viltolarsen

On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for viltolarsen, its investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. The program is open to all DMD patients . . .

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FDA Accepts NS Pharma’s NDA for Viltolarsen for the Treatment of DMD Amenable to Exon 53 Skipping

On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .

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NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping

On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its exon-skipping therapy under development, viltolarsen (previously NS-065/NCNP-01), to treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. On Sept. 26, Nippon Shinyaku . . .

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