Background: Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has extended the decision goal date for eteplirsen by a standard extension period of three months. The new date by which the FDA must make a decision about whether to approve eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) is May 26, 2016. . . .
Press Release: FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Summary: BioMarin reported today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) indicating that the review cycle for the company’s New Drug Application for drisapersen (Kyndrisa) to treat DMD is . . .
Researchers are looking for people with amyotrophic lateral sclerosis (ALS) to participate in the phase 3 VITALITY-ALS clinical trial, sponsored by Cytokinetics, to test the experimental drug tirasemtiv. Tirasemtiv is a skeletal muscle activator that is designed to increase the sensitivity of muscle fibers to calcium, which should cause these fibers to contract even if . . .
Please read the message below from Eli Lilly regarding the results of their phase 3 trial in Duchenne muscular dystrophy (DMD): Update on Phase 3 Tadalafil Trial We are writing to share disappointing news about our Phase 3 study of tadalafil in approximately 330 patients with Duchenne muscular dystrophy (DMD). We recently completed analysis of . . .
CAMBRIDGE, MA, and CHICAGO, IL, February 5, 2016 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, and the Muscular Dystrophy Association (MDA), today announced a collaboration to support the Part B portion of the MoveDMD clinical trial of CAT-1004, a novel product candidate for the treatment . . .
Jump to Storify Jump to Video The reviews are in! Last week’s event in New York City was a major hit! Check out some of these reactions from MDA’s Facebook followers: “I really enjoyed watching the live stream today of the event, As a person who has MD I want to Thank all those who . . .
Jordan Smith, winner of the ninth season of The Voice singing competition on NBC, closed out our January 29 event in New York with an emotional rendition of Miley Cyrus’s “The Climb.” The song’s lyrics — about obstacles and overcoming them despite great odds — resonated with our audience of families and supporters. Jordan, who once told a . . .
Thank you to everyone who participated in advocacy in 2015. You have each played an important role in bringing about policy changes that help save and improve the lives of kids and adults living with muscular dystrophy, ALS and related muscle-debilitating diseases. Through thousands of letters and emails to your members of Congress, in-person meetings . . .
Kasey Bennett loves football. In fact, it was during one of his games that his parents noticed there was something different about their young quarterback. “He couldn’t keep up with the other kids,” says his father. Soon after, Kasey was diagnosed with DMD. The disease has taken him out of the game but it hasn’t . . .
Zane Jacobs is a “wild and crazy” 7-year-old who “loves to get dirty and cause absolute chaos.” Though he is affected by DMD, he and his family are determined to make the most of their time together, whether that’s playing football in the front yard or swimming at the waterpark. When not horsing around with . . .
