April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

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March 28, 2019

Sarepta Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of ESSENCE Study

Sarepta Therapeutics announced positive results from its interim analysis of muscle biopsies at week 48, comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, randomized double-blind,…

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February 14, 2019

Sarepta Announces FDA Acceptance of Golodirsen NDA for People with Duchenne Amenable to Exon 53 Skipping

Sarepta Therapeutics, Inc. announced the FDA has accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of August 19, 2019. Golodirsen is a potential exon skipping…

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February 6, 2019

NS Pharma Begins Rolling NDA Submission to FDA for Viltolarsen (NS-065/NCNP-01)

PPMD is pleased to learn that NS Pharma has initiated submission of a rolling New Drug Application (NDA) seeking accelerated approval for viltolarsen (NS-065/NCNP-01), an investigational drug for the treatment of Duchenne in patients amenable…

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January 3, 2019

Wave Life Sciences Duchenne Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program

Wave Life Sciences announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative…

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December 20, 2018

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Golodirsen (SRP-4053)

PPMD is excited to learn that Sarepta Therapeutics, Inc.  announced that it has completed the submission of its rolling New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053), a phosphordiamidate morpholino oligimer engineered to treat…

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December 6, 2018

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

  Wave Life Sciences announced positive safety and tolerability results from the WVE-210201 Phase 1 clinical trial today. PPMD continues to be optimistic about the progress the team at Wave Life Sciences is making with…

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October 5, 2018

NS Pharma Announces Positive Results of US/Canada Phase II Clinical Trial of Viltolarsen at World Muscle Society

NS Pharma announced positive results of a US/Canada Phase II clinical trial of viltolarsen at the World Muscle Society meeting held in Mendoza, Argentina. The preliminary findings suggest clinical improvement as measured by timed function…

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September 18, 2018

Your Duchenne Registry Data: Researchers Find Different Mutations Have Varying Effects on Progression

As curators of The Duchenne Registry, we are frequently asking you to submit your genetic test report to the Registry. You may often wonder why we need the actual laboratory report, and why we are…

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August 16, 2018

Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201

  Wave Life Sciences today announced that the FDA has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne. These designations represent the significant progress being made…

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