August 16, 2018

Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201

  Wave Life Sciences today announced that the FDA has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne. These designations represent the significant progress being made…

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August 8, 2018

Connect with other Duchenne Families in Your Area

Online interactions are great, but nothing can replace in-person connections with other families living with Duchenne. PPMD has local Connect groups all over the country for this very reason. Have you “connected” with your state’s PPMD Connect group…

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August 6, 2018

Continuing to Enhance The Duchenne Registry

Many of you have probably noticed the new “look and feel” of The Duchenne Registry (previously DuchenneConnect) – see the end of this blog for a list of some of the new features! In honor of our…

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August 2, 2018

Mallinckrodt Announces First Patient Enrolled in Phase 2 Study of MNK-1411 in Duchenne Muscular Dystrophy

  PPMD is excited to learn that Mallinckrodt enrolled the first patient in their Phase 2 study assessing the efficacy and safety of the investigational drug MNK-1411 (cosyntropin, also known as tetracosactide). We look forward…

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July 25, 2018

Sarepta Therapeutics Announces Clinical Hold on Micro-Dystrophin Gene Therapy Trial

This afternoon Sarepta Therapeutics, Inc. announced that the micro-dystrophin Phase 1/2a gene therapy trial was placed on clinical hold by the FDA as the result of an out-of-specification production lot. The company was notified by the Research…

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July 24, 2018

WATCH: HOPE-2 Duchenne Clinical Trial – Webinar Recording with Capricor

Capricor Therapeutics recently joined PPMD for a community webinar update to discuss the HOPE-2 trial. During the webinar, Capricor provided a background of CAP-1002 and its clinical development to date that has led to the…

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June 27, 2018

Summit Therapeutics Announces PhaseOut DMD Did Not Meet Primary Endpoint

We are disappointed to learn that Summit Therapeutics plc today announced that their PhaseOut DMD trial has not met its primary or secondary endpoints after 48 weeks of treatment of ezutromid in patients with Duchenne….

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June 26, 2018

Vamorolone (VBP15) Top Line Data Shows Improvements in Strength and Endurance in Boys with Duchenne

ReveraGen BioPharma, with the Cooperative International Neuromuscular Research Group (CINRG), conducted an international clinical trial of vamorolone at 11 CINRG sites in 6 countries (US, Canada, Australia, Israel, UK, and Sweden). ReveraGen reports that the…

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June 19, 2018

Positive Preliminary Results from the First Three Children Dosed in Phase 1/2a Gene Therapy Micro-dystrophin Trial

Sarepta Therapeutics announced that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne. Ryan…

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June 18, 2018

Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001

PPMD is excited to learn that Solid Biosciences Inc. announced that the FDA has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001. In…

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