October 1, 2020

Pfizer Receives FDA Fast Track Designation for PF-06939926 Investigational Gene Therapy

Pfizer Inc. announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne has received Fast Track designation from the FDA. PF-06939926 is currently being evaluated to determine the safety and efficacy of…

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October 1, 2020

FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

PPMD is delighted to learn that the FDA has lifted the hold on Solid Biosciences’ IGNITE-DMD clinical trial for their gene therapy product (SGT-001) delivering a micro-dystrophin transgene via AAV9. Solid has worked diligently to…

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September 28, 2020

Catabasis Presents Information On Edasalonexent At 25th International Congress Of The World Muscle Society

Catabasis Pharmaceuticals today shared information on the edasalonexent program in Phase 3 development for Duchenne in poster presentations at the Virtual 25th International Congress of the World Muscle Society. The posters include new preclinical research…

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September 28, 2020

Sarepta Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001 Gene Therapy

PPMD is excited to share today’s announcement from Sarepta Therapeutics regarding two-year follow up results from four clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). Results presented at the Virtual 25th International Congress of the World…

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September 24, 2020

WATCH: Resiliency in Results – What We Learn When Trials Are Terminated (Webinar Recording)

No other community knows the importance of resiliency like the Duchenne community, as highlighted by our communal commitment to ensure that all people living with Duchenne have access to safe, accessible therapies. Clinical trials are…

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September 23, 2020

Santhera Announces Publication of Long-Term Clinical Data with Vamorolone in Patients with Duchenne

Santhera Announces Publication of Long-Term Clinical Data with Vamorolone in Patients with Duchenne Santhera announced a new publication in the journal PLOS Medicine describing new open-label, long-term clinical data on the safety, tolerability and efficacy…

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September 21, 2020

Join the Race to End Duchenne – Virtually!

Even though we’re apart, our community has never felt more together! Run (or walk, or roll) as one, by participating in PPMD’s first ever Race to End Duchenne Virtual Series. Choose from a 5K, 10K, or 15K…

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September 21, 2020

Pfizer Shares Update on PF-06939926 Gene Therapy Trial During Company’s Virtual Investor Day

Pfizer recently shared an update on their mini-dystrophin replacement gene therapy product PF-06939926 during their virtual Investor Day. In total 18 boys have received the gene therapy product as part of the Phase 1b study….

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September 20, 2020

New! PPMD’s Sibs Connect!

PPMD is launching our first-ever official group for siblings! If you have a sibling with Duchenne or Becker and are looking for a group to support you every step of this journey, this is for you. PPMD’s…

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September 18, 2020

Catabasis Shares September 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter.  Their plan is to share top-line results from the Phase 3 PolarisDMD trial of edasalonexent with the Duchenne community later this year. As highlighted in…

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