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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August 8, 2019

PPMD to Host Duchenne Platform Trial Community Meeting & Live Stream

Please join PPMD and the Duchenne community at an open meeting on September 9, 2019 to provide input into the protocol design for the Duchenne Platform Trial, with the aim to increase access to therapeutic…

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August 7, 2019

PPMD Announces Children’s Hospital of Richmond at VCU as 26th Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Children’s Hospital of Richmond at VCU (CHoR) the 26th Certified Duchenne Care Center (CDCC). This expanding certification program of elite centers, created in 2014 as part of PPMD’s…

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August 7, 2019

Audentes Therapeutics Announces Plans to Commence Patient Dosing with AT702 in Q4 2019

Audentes Therapeutics has announced plans to commence patient dosing in the fourth quarter of 2019 with AT702 produced by Nationwide Children’s Hospital. AT702 is designed to induce exon 2 skipping to treat Duchenne caused by…

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August 1, 2019

Race Athlete Spotlight: Tara Reynolds

Tara Reynolds has run the Aramco Houston Half Marathon with the Race to End Duchenne team for three years in a row, raising thousands of dollars. She does it all for her friend and inspiration,…

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