December 20, 2019

Sarepta Therapeutics Shares Winter 2019 Newsletter

Sarepta has shared the Winter 2019 issue of their newsletter, including updates on current Sarepta programs. Click here to download.

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December 16, 2019

Wave Life Sciences Announces Discontinuation of Suvodirsen Development for Duchenne

Wave Life Sciences Ltd. announced its decision to discontinue development of suvodirsen for patients with Duchenne who have mutations amenable to exon 51 skipping, based on its interim analysis of the Phase 1 open-label extension…

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December 13, 2019

FDA Grants Accelerated Approval to Vyondys 53

PPMD is excited to learn that the FDA has granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne patients who have a confirmed mutation of the dystrophin gene that is amenable to exon…

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December 4, 2019

Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics

Astellas Pharma and Audentes Therapeutics announced that they have entered into a definitive agreement for Astellas to acquire Audentes. We look forward to learning more as this partnership moves forward. Read the Announcement from Audentes…

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November 26, 2019

WATCH: NS Pharma Provides Viltolarsen Update – Webinar Recording

On November 20, 2019, NS Pharma joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of its investigational drug, viltolarsen (NS-065/NCNP-01), including its mechanism of action and regulatory status….

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October 22, 2019

WATCH: DYSTANCE 51, a Phase 2/3 Clinical Trial of Investigational Suvodirsen – Webinar Recording

Suvodirsen (formerly known as WVE-210201) is an investigational stereopure oligonucleotide being developed as a potential treatment for boys with Duchenne muscular dystrophy with mutations amenable to exon 51 skipping. Wave Life Sciences is a clinical-stage…

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October 2, 2019

NS Pharma Completes Rolling NDA Submission to FDA for NS-065/NCNP-01 (viltolarsen)

We are pleased to learn that NS Pharma has completed the submission of its rolling New Drug Application (NDA) to the FDA for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne amenable…

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September 16, 2019

Wave Life Sciences Announces Fast Track Designation from U.S. FDA for Suvodirsen

Wave Life Sciences shared today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to suvodirsen for the treatment of Duchenne in patients amenable to exon 51 skipping. Fast Track designation…

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September 5, 2019

Sarepta Therapeutics Shares Summer 2019 Newsletter

Sarepta has shared the Summer 2019 issue of their newsletter, including clinical updates. Click here to download.

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August 22, 2019

WATCH: Community Discussion with Sarepta Therapeutics – Webinar Recording

Yesterday PPMD invited Sarepta leaders to join a community discussion regarding the New Drug Application for golodirsen. We apologize if you were unable to access the webinar audio due to the high number of attendees….

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