September 13, 2018

Genentech/Roche RG6206 Community Letter

  Today, Roche provided the community on their clinical development program of RG6206, an investigational anti-myostatin adnectin that is designed to block the activity of myostatin. Roche also answers questions they have received in light…

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September 7, 2018

PPMD & Sarepta Partner to Launch Duchenne Outcomes Research Interchange

I am thrilled to announce PPMD’s plan to launch a patient- and physician-reported outcomes data warehouse – the Duchenne Outcomes Research Interchange (the Interchange) – which will combine data from The Duchenne Registry with clinician…

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August 30, 2018

Gene Repair Technology Restores Dystrophin Expression in Canine Model of Duchenne

We are encouraged to learn that Exonics Therapeutics, Inc., a biotechnology company focused on developing gene editing therapies to treat patients with Duchenne and other neuromuscular diseases, today announced the publication of a preclinical study…

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August 30, 2018

Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne

We are deeply disappointed to learn that Pfizer is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne: a Phase 2 safety and efficacy study (B5161002) and an open-label extension study…

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August 22, 2018

ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne

  ReveraGen BioPharma announced the initiation and first patient enrollment in a double-blind, placebo- and prednisone-controlled clinical trial (VBP15-004) of vamorolone, a first-in-class dissociative steroidal anti-inflammatory, in Duchenne. This VISION-DMD study will enroll approximately 120…

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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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