… is to raise awareness of Duchenne muscular dystrophy, a progressive disorder affecting 1 … Ryan had been diagnosed with Duchenne muscular dystrophy in December 2007. The Schultzes … research into a cure or treatment for DMD. The nonprofit will …

… biotech won approval in 2016 for Exondys 51 (eteplirsen), its Duchenne muscular dystrophy (DMD) drug … atrophy drug Spinraza, and Jazz Pharmaceuticals… ; hepatic veno-occlusive disease drug Defitelio. This thumbs-down from the FDA …

… second drug that aimed to treat children with Duchenne muscular dystrophy, a … the development of medicines for unmet medical needs. The … to confirm treatment benefit in a later clinical trial. Sarepta … clinical trials required by the FDA to confirm the drug’s …

… , August 20, 2019 Boston Globe Pharmaceutical company Sarepta Therapeutics dropped as … rejection of its second drug for Duchenne muscular dystrophy. The FDA cited risk of infection … " of Sarepta's treatment, according Cantor analysts. Watch Sarepta …

… experimental drug to treat patients with Duchenne muscular dystrophy. … Sarepta SRPT, -14.44% disclosed on Monday afternoon that the FDA … drug first, and the company would do a trial … . More on Duchenne trials: Sarepta Therapeutics …

… for golodirsen injection for treating Duchenne muscular dystrophy, or DMD, in patients with … as a surprise, as the drug watchword had earlier in 2016 … . Related Links: The Daily Biotech Pulse: FDA Likes Nabriva's Antibiotic …

… FDA rejection of Vyondys 53, the company’s latest treatment for Duchenne Muscular Dystrophy … with the FDA. Using a similar mechanism, this drug skips exon …   to the FDA from an open-label trial of 25 … boys with DMD, which showed signs of the drug …

… drug, Exondys 51 – which targetings the deadly muscle-wasting disease Duchenne Muscular Dystrophy … medicines could have a long-term impact after just one treatment … trials and may require some degree of FDA … Opinion columnist covering biotech, pharma and health …

… day for a few biotech companies that saw either … branded and generic opioid medications. Under the settlement in … U.S. Food and Drug Administration (FDA). Essentially, the firm … golodirsen injection for the treatment of Duchenne muscular dystrophyin patients …