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 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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August 14, 2019

Solid Biosciences Provides Update on IGNITE DMD Clinical Trial

Today, Solid announced that the second patient has been dosed in the higher dose cohort of the IGNITE DMD Phase I/II clinical trial of SGT-001, the company’s AAV gene transfer candidate under investigation for Duchenne….

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August 8, 2019

Sarepta Therapeutics Comments on Erroneous Submission to US FDA Adverse Event Reporting System (FAERS)

Sarepta Therapeutics was informed earlier today that an adverse event report was submitted to the FDA’s adverse event reporting system (FAERs), a post-marketing surveillance database for approved therapies.  Sarepta states that based on their investigation…

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