Highlights from day three at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly.  Mallinckrodt Pharmaceuticals Mallinckrodt provided an update on their Phase 2 multicenter, double-blind, placebo-controlled study, assessing the safety and efficacy of MNK-1411 in Duchenne patients. MNK-1411 is an injectable synthetic 24-amino acid peptide analogue of adrenocorticotrophic […]
Highlights from day two at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly. Sarepta’s Gene Therapy Program: Sarepta provided a much-anticipated update on their dystrophin mini gene clinical study. The latest data release reinforced the positive news reported earlier for the first three patients. Today’s data — for […]
Highlights from the first day at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly.  Carrier-mothers: It’s recognized that Duchenne and Becker muscular dystrophy carrier-mothers are at high risk for developing various dystrophic complications. Recent studies suggest that the incidence of cardiac and non-cardiac manifestations occurs earlier (and with […]
Dear Friends, Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and […]
Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing in early scientific research to accelerate a pathway to a cure for Duchenne, CureDuchenne is committed to providing the Duchenne community with education, help and […]
Dedicated Online Platform DuchenneXchange.org Launches to Unite, Support and Educate Duchenne Community Collaborative site offers a trustworthy, moderated online space where Duchenne families, caregivers, NGOs and pharmaceutical companies can connect and share knowledge NORWALK, CT (July 31, 2018) — The Duchenne muscular dystrophy (DMD) community now has a dedicated online platform where they can connect […]
PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]
The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was […]
The second day of the conference focused on gene editing, gene therapy as well as the latest research into the development of disease biomarkers. Presentations included: Dongsheng Duan (University of Missouri) described his research groups efforts that led to the selection of an optimized micro dystrophin gene construct for systemic AAV deliver to Duchenne patients. […]
CureDuchenne staff is participating in the New Direction in Biology and Disease of Skeletal Muscle Conference in New Orleans this week.  Our staff scientist, Michael Kelly, will be providing a summary of key learnings each day.  Have a question?  Leave us a comment and Michael will respond. Jane Owens (Rare Disease Research Unit, Pfizer) provided an […]
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