Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of dystrophin — the protein lacking in Duchenne muscular dystrophy (DMD) — and improves motor abilities in boys with the disorder, according to updated data from a Phase 1b trial. “Based on the…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the disease, top-line data from the HOPE-2 clinical trial show. “I am incredibly pleased with the outcome of the…
The U.S. Food and Drug Administration (FDA) is requesting more information on the process used to manufacture the gene therapy SGT-001, as it reviews the clinical hold placed on the Phase 1/2 trial of this Duchenne muscular dystrophy (DMD) therapy candidate, Solid Biosciences reported. IGNITE DMD (NCT03368742) will remain…
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
People with Duchenne and Becker muscular dystrophy should not forego their routine health assessments during the COVID-19 pandemic, but are encouraged to transition their care to telemedicine and to medical tests performed at home to minimize risk. Those are among the consensus recommendations provided by neuromuscular specialists to help these patients…
CureDuchenne Ventures has given $1 million in seed funding to support the development of Myosana Therapeutics‘ potential non-viral gene therapy for people with Duchenne muscular dystrophy (DMD). The money is expected to advance a platform that delivers the full DMD gene to the muscles…
Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar hosted by Parent Project Muscular Dystrophy (PPMD). PTC is the maker of Emflaza…
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…
FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…
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