December 6, 2022

Complete Your Registry Surveys and You Could Win Your Choice of a $150 Holiday Gift Card!

It’s the Holiday Season and we are thrilled to have another great raffle for families who participate in The Duchenne Registry! This December, the Registry will be giving away TWO $150 e-gift cards to the…

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December 5, 2022

Solid Biosciences Announces Completion of AavantiBio Acquisition & Update on Duchenne Pipeline Program

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have completed the acquisition of AavantiBio, a gene therapy company. The combined company will focus on advancing a…

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November 28, 2022

Sarepta Announces Biologics License Application (BLA) Accepted For Filing by the FDA for SRP-9001

PPMD is thrilled to learn that the FDA has accepted for filing and granted priority review for the Biologics License Application (BLA) for Sarepta’s gene therapy product, SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant…

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November 21, 2022

PTC Shares Community Letter Updating on Activities To Bring Ataluren (Translarna) To US Patients

PTC Therapeutics has shared a community letter updating on the company’s activities to bring ataluren (Translarna) to US patients with nonsense mutations. PPMD will host a community webinar with PTC on Friday, December 9th at…

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November 18, 2022

Pfizer Announces First Participant Dosed in DAYLIGHT Phase 2 Gene Therapy Trial

Pfizer has announced that the first participant has been dosed in the Phase 2 DAYLIGHT study, which will evaluate the safety and tolerability of the company’s investigational mini-dystrophin gene therapy product (fordadistrogene movaparvovec) in very young…

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November 8, 2022

Astellas Announces Decision to Discontinue Screening & Enrollment Activities for ASP0367 (MA-0211)

PPMD is disappointed to learn that Astellas has announced its decision to discontinue further screening and enrollment activities for ASP0367 (MA-0211) in participants with Duchenne. This decision was not based on safety concerns, but due…

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October 21, 2022

WATCH: Edgewise Therapeutics – A Novel Approach to Protect Muscle in Duchenne & Becker (Webinar Recording)

PPMD was joined by Edgewise Therapeutics for a community webinar on October 19, 2022. Edgewise’s Chief Scientific Officer and Chief Medical Officer discussed the clinical development program for EDG-5506, a myosin modulator, in Becker and…

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October 18, 2022

Edgewise Announces 6 Sites Enrolling for CANYON, Phase 2 Trial of EDG-5506 in Becker

Edgewise has shared a community letter announcing that six sites are now open for enrollment for the Canyon trial for individuals living with Becker muscular dystrophy. Canyon is a Phase 2 trial of EDG-5506, a…

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October 17, 2022

The Duchenne Registry has three new surveys!

The Duchenne Registry, powered by PPMD, is the largest, most comprehensive patient-reported registry for Duchenne and Becker muscular dystrophy. With over 5,000 registrants, the Registry connects Duchenne and Becker patients with actively recruiting clinical trials…

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September 30, 2022

Solid Biosciences Acquires AavantiBio & Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Biosciences shared an update with the Duchenne community regarding their strategic development. Solid announced they have acquired AavantiBio, a gene therapy company with a focus on rare cardiomyopathies and Friedeich’s ataxia. While this…

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