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. . . .
Kevin Clay is going to the Super Bowl. A Carolina Panthers superfan, he will be in California this weekend to cheer on his beloved team. The opportunity to see the big game in person is, as Kevin says, “a dream come true.” But the trip to Levi’s Stadium in Santa Clara, Calif., is particularly meaningful . . .
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 . . .
Like most recent college graduates preparing to enter the “real world,” I was in the throes of the inevitable what-the-heck-am-I-going-to-do-with-my-life panic when an unexpected call set me on a new path. In accepting the offer to become the first adult MDA National Goodwill Ambassador, I could stop speculating about what my next life journey will . . .
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 . . .
If you are interested in muscular dystrophy research, you need to know about the MDCC… What is the MDCC? You may have previously come across the acronym “MDCC” posted on MDA’s website or social media pages. MDCC stands for the Muscular Dystrophy Coordinating Committee. The Committee was established under the MD-CARE . . .