December 23, 2019

Sarepta Announces Partnership with Roche Outside the U.S. for its Investigational Micro-dystrophin Gene Therapy

Sarepta Therapeutics, Inc. today announced that Sarepta and Roche have entered into a licensing agreement providing Roche exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for Duchenne muscular dystrophy, outside the United States….

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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 20, 2019

WATCH: Better Todays. Stronger Tomorrows. – Webinar Recording

On December 19, 2019, PPMD had the privilege of hosting a conversation with Duchenne experts on the importance of optimal care in Duchenne, how care impacts every aspect of our mission to end Duchenne and our…

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December 20, 2019

Winter Safety & Bone Health

With the official start of winter quickly approaching and the icy weather already here, we want to remind your family about the importance of safety, and also what to do if and when accidents happen….

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December 19, 2019

Mallinckrodt Terminates BRAVE Study Investigating the Efficacy and Safety of MNK-1411 in Duchenne

PPMD is disappointed to learn that Mallinckrodt has made the decision to terminate their BRAVE Study (MNK14112096). Based on the company’s statement below, the decision appears to have been the result of low enrollment and…

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December 18, 2019

PPMD Awards $465,000 Grant to The Ohio State University to Rescue Cardiac Dystrophin & Function in Duchenne

Parent Project Muscular Dystrophy is excited to announce that Renzhi Han, an associate professor at The Ohio State University College of Medicine, will receive a $465,000 grant to study the potential use of in vivo…

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December 18, 2019

Solid Biosciences Provides Data Update from SGT-001 Development Program

Solid Biosciences Inc. provided an update on the IGNITE DMD phase I/II microdystrophin gene therapy clinical program. Solid shares that the status of the sixth patient dosed in the higher 2E/14 vg/kg, who experienced the…

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December 17, 2019

Invest today, improve tomorrow. ALL DONATIONS MATCHED.

I am a clinical psychologist specializing in child behavior and I’m married to an incredible woman who is a pastor. We have made it our life’s mission to help other people. In 2006, our world…

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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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