PTC Therapeutics reported today the U.S. Food and Drug Administration’s Office of Drug Evaluation is unable to approve the company’s New Drug Application for ataluren (brand name Translarna) for the treatment of Duchenne muscular dystrophy (DMD) caused by a premature stop codon, or nonsense mutation, in its current form.
The FDA yesterday convened a meeting with the PCNS, to review the NDA, for ataluren (brand name Translarna) to treat some forms of DMD. Following a presentation of the data in support of the NDA by PTC Therapeutics the overwhelming majority (10 out of 11) suggested that although it is possible that Translarna may be effective, the data are inconclusive and more work is needed to determine whether, in fact, it is.
The FDA on Thursday will convene a meeting with the Peripheral and Central Nervous Systems Drugs Advisory Committee (PCNS), to review the new drug application, or NDA, for ataluren (brand name Translarna) DMD.
Sarepta announced encouraging results from a phase 1/2 DMD clinical trial for golodirsenm which targets exon 53.
Dr. Mattia Quattrocelli of Northwestern University was awarded an MDA grant to study the effects of glucocorticoids in DMD.
In recognition of World Duchenne Awareness Day we share our support w all DMD families, including Stacey Hudak and her son Ryan, who lives w DMD.
Learn about our most recently awarded research grants and the DMD families they have the potential to impact.
Tejvir Khurana, a professor at the University of Pennsylvania was awarded an MDA grant to test a therapeutic strategy for DMD.
Postdoctoral associate James Novak was awarded an MDA grant totaling $180,000 to study exon skipping drugs in DMD.
In October 1986 Jerry Lewis announced the DMD gene had been discovered, a monumental moment in MD research.
