Muscular Dystrophy News

March 15, 2018March 15, 2018 News TheU.S. Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial testingSGT-001, a dystrophin gene therapy intended to treat Duchenne muscular dystrophy (DMD), after the ... Read more March 14, 2018 News Sarepta Therapeuticswill seek accelerated U.S. approval of a treatment for a subset ofDuchenne muscular dystrophy (DMD) patients. Golodirsen (SRP-4053)is for patients whose disease stems from a deleted part of ... Read more March 14, 2018 News When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress SMARD, for short. Hunter Pageau, a 12-year-old boy from ... Read more March 13, 2018March 12, 2018 columns, You, Me and MD - a Column by Leah Leilani Being wheelchair-bound and still stylish can be the ultimate challenge. Its hard to find that balance between style and comfort, especially as a woman. The average, able-bodied female millennial ... Read more March 12, 2018 News In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new ... Read more March 12, 2018March 12, 2018 News Research supporting prenatal diagnosis of Duchenne muscular dystrophy (DMD) has earned Vietnamese scientist Tran Van Khanh a Kovalevskaya Award. Khanh has focused her work on studying gene therapy and ... Read more March 9, 2018 News Summit Therapeuticshas opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy(DMD) who participated in the previous Phase 1 clinical ... Read more March 9, 2018 News At a time of unprecedented polarization in Congress, two U.S. lawmakers one Republican, one Democrat are stressing the urgency of working across the aisle to help the estimated ... Read more March 7, 2018March 7, 2018 News Plasticity Brain Centershas become an authorized care provider for children and young adults with muscular dystrophyand other rare disorders who receiveGardiner Scholarshipsfrom the State of Florida. In addition to ... Read more March 7, 2018March 7, 2018 columns, You, Me and MD - a Column by Leah Leilani Ive had bad posture my entire life. Growing up, I didnt know why I always slouched so much. I just knew that I was comfortable that way. Years later, ... Read more March 5, 2018March 5, 2018 News A Phase 2 clinical trial ofATL1102, Antisense Therapeuticsinvestigational therapy for Duchenne muscular dystrophy (DMD),recently received authorization to proceed from theRoyal Childrens Hospital (RCH),Melbourne Human Research Ethics Committee. ATL1102 inhibits ... Read more March 2, 2018 News Retrophin and the U.S. subsidiary of Britains Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Childrens National Health Systemin ... Read more

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Muscular Dystrophy News