research

Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada

posted on March 17, 2015 - 2:31pm
BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).

MDA-supported researcher Charles Gersbach and colleagues have developed a strategy to change dystrophin DNA that has shown promise in the lab

posted on February 26, 2015 - 11:30am
MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb.

This report on treatment development for Duchenne muscular dystrophy contains news about fighting fibrosis (scarring), treating DMD-related heart disease, exon skipping and the corticosteroid deflazacort

posted on February 2, 2015 - 12:04pm
Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene. Fighting fibrosis

Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research

posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?

Meet Grace Pavlath, MDA's new scientific director

posted on January 5, 2015 - 9:09am
Quest Winter 2015
Grace Pavlath, an accomplished muscle disease researcher and a professor in the Department of Pharmacology at Emory University School of Medicine in Atlanta, recently joined MDA as senior vice president and scientific program director. She will be based in Atlanta initially and will eventually relocate to Washington, D.C.

Research updates and clinical trials information

posted on January 5, 2015 - 9:07am
Quest Winter 2015
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.

PTC Therapeutics has begun submitting an application for U.S. approval of Translarna (ataluren) for Duchenne MD; completion of the application is slated for late 2015

posted on December 23, 2014 - 11:20am
New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.

In this second of two fall reports on treatment development for Duchenne muscular dystrophy, news about ARM210, CAP-1002, PDE5 inhibitors, drisapersen and CAT-1004 is presented

posted on December 4, 2014 - 3:13pm
  Update (Feb. 6, 2015): This article has been amended to show that Catabasis' compound CAT-1004 is expected to move into clinical testing in boys with DMD during the first half of 2015. =======================================================================================================================