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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Wave Life Sciences Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for Therapies Amenable to Skipping Exons 51 and 53

Today, Wave Life Sciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program for suvodirsen, its therapy for patients amenable to exon 51 skipping, and its therapy for patients amenable to exon 53 skipping. The full letter follows. Dear Duchenne community: We have a disappointing update . . .

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Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

On Jan. 3, Wave Life Sciences announced that its upcoming Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, has been selected by the U.S. Food and Drug Administration (FDA) for its complex innovative trial designs (CID) pilot program.  Wave . . .

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Wave Life Sciences Announces Positive Results of Phase 1 Trial of WVE-210201 for Duchenne Muscular Dystrophy

Today, Wave Life Sciences announced that its Phase 1 trial of WVE-210201, an investigational, exon skipping therapy for boys with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, showed positive safety and tolerability data. Based on these results, Wave announced that it will initiate a Phase 2/3 clinical trial of WVE-210201 in 2019. Wave . . .

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