September 18, 2018

Your Duchenne Registry Data: Researchers Find Different Mutations Have Varying Effects on Progression

As curators of The Duchenne Registry, we are frequently asking you to submit your genetic test report to the Registry. You may often wonder why we need the actual laboratory report, and why we are…

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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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