Muscular Dystrophy News – Muscular Dystrophy News

May 30, 2018May 30, 2018NewsShould scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of ... Read moreMay 30, 2018May 30, 2018NewsResearchers have generated skeletal muscle from human stem cells in the laboratory, an achievement that may advance development of treatments and tissue replacement for patients with muscular dystrophy (MD). ... Read moreMay 29, 2018May 29, 2018columns, You, Me and MD - a Column by Leah LeilaniJust eight years ago, a four-hour trip to Disneyland would barely touch me. Id go on a few rides, have a bite to eat, come home, take a nap, ... Read moreMay 29, 2018May 29, 2018NewsFinding treatments and potential cures for rare diseases is crucial, but so is the quality of patients lives a rather nebulous term that means different things to different ... Read moreMay 25, 2018May 25, 2018NewsDaiichi Sankyoannounced positive safety findings in a Phase 1/2 trialof its investigational dystrophin gene therapyDS-5141in a small group of Duchenne muscular dystrophy (DMD) patients, but noted results failed to ... Read moreMay 24, 2018NewsTwo years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation(GDPR) a tough new law that aims to protect the EUs 512 ... Read moreMay 23, 2018May 23, 2018NewsSarepta Therapeutics and Invitae, a company specializing in genetic diagnostics,are expanding a partnership to help clinicians more quickly identify people withDuchenne muscular dystrophy (DMD). Sarepta is working to advance ... Read moreMay 22, 2018May 21, 2018columns, You, Me and MD - a Column by Leah LeilaniEveryone suffers from insecurity every once in a while. It doesnt matter whether youre a man or a woman, a child or a teenager. Nor if youre disabled or ... Read moreMay 21, 2018May 21, 2018NewsThe U.S. Food and Drug Administration recently grantedrare pediatric disease designation toMyonexus Therapeutics experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or ... Read moreMay 18, 2018May 18, 2018NewsTheU.S. Food and Drug Administration (FDA)has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapys developer, announced. The company also has filed ... Read moreMay 17, 2018May 17, 2018NewsThe National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORDs founding at a dinner tonight in Washington, D.C. ... Read moreMay 16, 2018NewsThe European Union isnt doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in ... Read more

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