September 10, 2019

Event Spotlight: Houston’s Coach To Cure MD

I have had so many people calling to ask “what can I do for Coach To Cure MD?” so I thought I would talk about our longest running Coach To Cure MD event in Houston…

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September 9, 2019

Decode Duchenne: Five Years of Free Genetic Testing

For over five years, PPMD has been proud to offer free genetic testing, counseling support and education through Decode Duchenne. This program is collaborative partnership between PPMD, industry partners Sarepta Therapeutics and PTC Therapeutics and…

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September 6, 2019

Watch Live: Duchenne Platform Trial Community Meeting

Parent Project Muscular Dystrophy (PPMD) will co-host the first Duchenne Muscular Dystrophy Platform Trial Community Meeting, along with collaborators I-ACT for Children and Critical Path Institute (C-Path) on September 9, 2019 at the Sheraton Silver…

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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 28, 2019

Join Our Team and Tackle Duchenne!

Coach To Cure MD, presented by Werner Ladder, is back for its 12th season ONE MONTH from today on September 28! Join our team and help #TackleDuchenne. Whether planning your own fundraising event or joining a…

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August 27, 2019

Santhera Announces Publication by ReveraGen of Positive Phase IIa-Extension Study Results with Vamorolone

A publication in the journal Neurology was released today describing efficacy studies of vamorolone, a treatment option being evaluated as a potentially safer alternative to corticosteroids, in people with Duchenne. PPMD is pleased to learn…

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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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August 19, 2019

Sarepta Therapeutics Receives Complete Response Letter from the FDA for Golodirsen New Drug Application

PPMD is very disappointed to learn that Sarepta Therapeutics received a Complete Response Letter from the FDA regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne in…

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August 16, 2019

ICER Releases Report; PPMD Issues Open Letter to Payers

Last night, the Institute for Clinical and Economic Review (ICER) released their final report assessing three Duchenne products: Emflaza, EXONDYS 51, and golodirsen. This report integrates all public comments, stakeholder engagement, and the Roundtable proceedings…

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August 14, 2019

Catabasis Shares August 2019 Newsletter

Catabasis shares the latest edition of their community newsletter, including an update on the Phase 3 PolarisDMD trial which is enrolling boys affected by Duchenne ages 4 to 7, any mutation type, that are not…

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