Today we couldn’t be happier as, together with the Duchenne community, we celebrate FDA accelerated approval of eteplirsen to treat kids and adults with some forms of Duchenne muscular dystrophy (DMD). “This is something that we’ve watched, and hoped and prayed for, and seen it develop over the last seven years or so,” said Josh . . .
Osaka-based Mitsubishi Tanabe Pharma announced today that its New Drug Application (NDA) for the drug edaravone to treat people with ALS (amyotrophic lateral sclerosis) was accepted by the U.S. Food and Drug Administration. The Japanese pharmaceutical company said it expects a decision on whether it can market the drug in the United States by June 16, 2017. . . .
Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has requested that the company provide dystrophin data from biopsies that were taken as part of the ongoing confirmatory study of eteplirsen, called PROMOVI. The data was requested by the agency in connection with its ongoing evaluation of Sarepta’s New Drug Application (NDA) . . .
The FDA has made available background materials and webcast information for the advisory committee meeting to review eteplirsen on Monday, April 25. To view the complete set of background information including a meeting agenda, meeting roster, committee roster and briefing information, visit 2016 Meeting Materials, Peripheral and Central Nervous System Drugs Advisory Committee. The Center . . .
CHICAGO, Feb. 16, 2016 — Lowe’s stores nationwide unite to participate in the Muscular Dystrophy Association’s iconic Shamrocks program now through March 31. This year marks 15 years of Lowe’s Shamrocks support that helps improve lives in communities across the country and build stronger futures for kids and adults with muscular dystrophy, ALS and related . . .
#MDAshamrocks ATLANTA, March 6, 2015 — Kroger-Atlanta Division stores will raise money this spring to help the Muscular Dystrophy Association save and improve the lives of people fighting muscle disease by participating in the 33rd annual MDA Shamrock program. From March 8 through March 21, Kroger customers and associates can help accelerate progress in . . .
New Berlin fourth-grader continues to advocate for MDA through historic awareness program CHICAGO, Jan. 8, 2015 — Reagan Imhoff spent 2014 jet-setting across the country, capturing the hearts of many as the face and voice of the Muscular Dystrophy Association — and now the 9-year-old will return as the 2015 National Goodwill Ambassador. Video: Meet Reagan . . .
Background: PTC Therapeutics yesterday announced results from the phase 3 “ACT DMD” trial of ataluren, an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The trial results showed clinically meaningful benefits for ataluren-treated patients, indicating the drug may slow functional decline in DMD patients, with some patients apparently . . .
Legislation Helps Ensure Access to Clinical Trials for People with Rare Diseases On October 7, 2015, President Obama signed into law the Ensuring Access to Clinical Trials Act of 2015 (S. 139/H.R. 209) (EACT), which removes a barrier to participating in rare disease clinical trials. EACT makes permanent a law that allows individuals to participate . . .
Background: Sarepta Therapeutics reported today additional long-term clinical efficacy and safety data from its pivotal phase 2b program of eteplirsen to treat Duchenne muscular dystrophy (DMD). The data demonstrated that over a three-year period of time, trial participants who were treated with eteplirsen experienced a slower rate of decline and were, on average, able to . . .