October 26, 2020

Antisense Therapeutics Receives US FDA Orphan Drug Designation for ATL1102 for the Treatment of Duchenne

Antisense Therapeutics announced today that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to the ATL1102 for treatment of Duchenne muscular dystrophy. ATL1102 is an antisense drug aimed at reducing…

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October 23, 2020

Solid & Ultragenyx Announce Strategic Collaboration to Develop & Commercialize New Gene Therapies for Duchenne

We are pleased that Solid Biosciences has announced a strategic collaboration and license agreement with Ultragenyx Pharmaceutical. Emil Kaakis, CEO of Ultragenyx, has been focused on rare diseases across his career and has considerable experience…

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October 12, 2020

Italfarmaco Receives FDA Rare Pediatric Disease Designation & Completes EPIDYS Phase 3 Trial Enrollment

Italfarmaco has provided an update on the development of Givinostat, sharing that the FDA has granted a Rare Pediatric Disease designation to Givinostat for the treatment of Duchenne, which allows an expedited review process for…

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October 8, 2020

Sarepta Therapeutics Shares Fall 2020 Newsletter

Sarepta has shared the Fall 2020 issue of their newsletter, providing updates on community programs and highlighting their new class of Route 79 scholarship recipients. Click here to download.

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October 6, 2020

Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa® in Duchenne

Today, we are disappointed to learn that Santhera announced its decision to discontinue development of its Phase 3 SIDEROS study with Puldysa® (idebenone) in Duchenne. Santhera will stop the SIDEROS trial (including extension) and participants…

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October 2, 2020

Catabasis Shares October 2020 Newsletter

Catabasis Pharmaceuticals has shared the latest edition of their community newsletter which includes information from their presentations at the Congress of the World Muscle Society. The Phase 3 PolarisDMD trial and GalaxyDMD open-label extension trial…

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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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