PPMD is proud to share our partnership with the Cooperative International Neuromuscular Research Group (CINRG) to advance the expanded Duchenne Natural History Study (eDNHS) study. Joining PPMD in support of the eDNHS study is Insmed Gene Therapy, LLC; ITF Therapeutics, LLC; NS Pharma, Inc; Santhera Pharmaceuticals (Switzerland), Ltd; Sarepta Therapeutics, Inc; Solid Biosciences Inc; TRiNDS, LLC; and the University of California, Davis.
The eDNHS is an expanded continuation of the previous Duchenne Natural History Study, which ran from 2005-2016. The study is sponsored by the University of California, Davis and is active and enrolling at ten sites world-wide and includes sites in the United States (6), Canada (2), Australia (1) and India (1). The data resulting from this long-term observational study in individuals with Duchenne muscular dystrophy (DMD) benefits the Duchenne community by facilitating development and approval of novel therapies and improving global clinical practice and policy guidelines.
“We are thrilled that PPMD and six sponsors in the Duchenne therapeutic development space have joined together to advance our understanding of how Duchenne progresses in boys and men with Duchenne,” explains Craig McDonald, MD, one of the eDNHS Study Chairs and University of California, Davis Co-Principal Investigator. “As more therapies become available to those living with Duchenne it is critical we collect data to understand the therapeutic impact and how to implement best care for our families.”
Boys and men with Duchenne are eligible to participate in the eDNHS regardless of functional status, participation in active or past clinical trials, or current or past therapies and medications. Study visits take place approximately every six (6) months to align with standard of care visit schedules and include questionnaires, medical history and medication review, and strength and function testing.
To share more about the study, PPMD will host a community webinar on Wednesday, April 1st from 1:00-2:00PM EST. Registration for the webinar is available here, and you can submit questions in advance here.
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