January 3, 2020

Thank you for making today better!

We can’t begin to express our gratitude to everyone who donated to PPMD this holiday season! Thanks to your generosity, and a community match, I am thrilled to announce that we reached our ambitious fundraising…

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December 27, 2019

Donate to PPMD today and help make tomorrow TWICE as strong

I know we are right in the thick of the holiday season with a new year quickly approaching, so I thank you for taking a minute to read this note. The holidays can be a…

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December 23, 2019

Advocacy Matters! Duchenne Community Requests Included in 2020 Federal Budget

Bipartisan agreement reached on funding bill includes Duchenne specific funding levels and report language advocated for by PPMD advocates! Who says Congress can’t agree on anything? After 147 in-person meetings in Washington DC by Duchenne…

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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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