FDA Approves First-Ever Treatment of SMA
The U.S. Food and Drug Administration (FDA) has approved Biogen’s nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA).
Tag: Breaking News
Eteplirsen Granted Approval for DMD: Turning Promise into Progress
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 . . .
NDA Filing for ALS Treatment Accepted by FDA
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. . . .
FDA Has Requested Additional Data for Eteplirsen Review
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) . . .
Eteplirsen Advisory Committee Meeting: Background Materials and Webcast Information
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 . . .
Lowe’s Supports MDA Shamrocks Program and Summer Camps to Help Families and Children with Muscular Dystrophy
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 . . .
Kroger-Atlanta Division Supports MDA Shamrock Tradition to Help Families Fighting Muscle Disease
#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 the . . .
Wisconsin Girl Returns as MDA National Goodwill Ambassador in 2015
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 . . .
PTC Announces Phase 3 Trial Results for Ataluren in DMD
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 . . .
President Obama Signs Ensuring Access to Clinical Trials Act (S. 139/HR209) Into Law
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 . . .
