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WATCH: Introducing PPMO – Webinar Recording with Sarepta Therapeutics

Sarepta Therapeutics, Inc. recently joined Parent Project Muscular Dystrophy (PPMD) for a community webinar update to discuss PPMO, Sarepta’s next generation precision RNA-targeting therapeutic platform for use in Duchenne muscular dystrophy. During the webinar, Sarepta…

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WATCH: Anti-Myostatin Adnectin RG6206 in Duchenne – Webinar Recording with Roche/Genentech

Roche/Genentech recently joined Parent Project Muscular Dystrophy for a webinar to provide an update on their Anti-Myostatin Adnectin (RG6206) development program. The team at Roche and its US affiliate, Genentech, and provided an overview of…

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Santhera Pharmaceuticals Launches Formal Expanded Access Program in the U.S.

PPMD is pleased to learn that Santhera Pharmaceuticals has launched a formal expanded access program in the U.S. for their investigational therapies, including idebenone. Learn more by visiting the Santhera website.

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May 16 Webinar: Roche/Genentech Anti-Myostatin Adnectin RG6206 in Duchenne

Roche/Genentech will join Parent Project Muscular Dystrophy for a webinar on Wednesday, May 16 at 1:00 PM EDT to provide an update on their Anti-Myostatin Adnectin (RG6206) development program. You will learn more about the team at…

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May 9 Webinar: Introducing PPMO – The Future of Precision RNA-Targeted Therapies for Duchenne

Sarepta Therapeutics, Inc. will join Parent Project Muscular Dystrophy for a webinar update on Wednesday, May 9 at 1:00 PM EDT to discuss PPMO, Sarepta’s next generation precision RNA-targeting therapeutic platform for use in Duchenne…

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PPMD’s Duchenne Registry Reaches 10-Year Milestone

PPMD is thrilled to announce that DuchenneConnect is now The Duchenne Registry. We recently reached a significant milestone of collecting 10 years worth of patient-reported data, and YOU have made this possible! Thank you for…

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[WEBINAR] What is a Master Protocol?

PPMD fights to end Duchenne for every single family. As part of our mission, we believe that we must continually work to identify and explore innovative opportunities that may accelerate the drug development and clinical…

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January 10, 2018

First Duchenne Patient Dosed in Microdystrophin Gene Therapy

“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM” On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr….

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January 8, 2018

PPMD’s Certified Duchenne Care Center Program – 2017 Impact & Progress

PPMD’s Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the CDC Care Considerations, available and accessible to as many families as possible, and to strengthen clinical trial…

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January 3, 2018

How You Can Report Serious Drug Side Effects to the FDA

When our children are hurting, we go to our medicine cabinets and take great care to select products that will ease their pain. Reduce the fever. Relieve the ache. In Duchenne, we know that the…

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