Dear MDA family and friends, At yesterday’s all-day FDA advisory committee meeting for the promising drug eteplirsen, MDA stood shoulder to shoulder with our sister groups, research and clinical experts, and most importantly you, our families. We couldn’t have been more proud to raise our voice on behalf of the thousands of Duchenne muscular dystrophy . . .
Today, MDA’s Executive Vice President & Chief Medical and Scientific Officer Dr. Valerie Cwik spoke during the FDA’s Advisory Committee hearing to review eteplirsen, under development by Sarepta Therapeutics for the treatment of some forms of Duchenne muscular dystrophy (DMD). Good afternoon. I am Dr. Valerie Cwik, and I am pleased to be here today on behalf . . .
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 . . .
Researchers are looking for boys with Duchenne muscular dystrophy (DMD) to participate in the ongoing phase 3 clinical trial, Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR-DMD). The trial will compare three different corticosteroid regimens in boys with DMD, ages 3 to 7 years. Corticosteroids, such as prednisone and deflazacort, work as anti-inflammatories or . . .
Antoni Barrientos, professor of neurology and of biochemistry and molecular biology at the University of Miami Miller School of Medicine in Miami, Fla., was awarded an MDA research grant totaling $300,000 over a period of three years to address a knowledge gap about mitochondrial protein complex assembly defects, which are a frequent cause of inherited . . .
ReveraGen BioPharma has announced that it has received a $3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health to conduct phase 2 clinical trials of vamorolone (previously VBP15) in boys with Duchenne muscular dystrophy (DMD). Vamorolone, a “dissociative steroid,” is an anti-inflammatory compound. Researchers hope . . .
The more I do with MDA, the more I learn about all that MDA is doing behind the scenes for our families. The 2016 Clinical Conference is a great example. The conference convenes world-renowned medical professionals to share best practice and discuss research advances — all for the benefit of MDA families. To attend as . . .
Chris Lorson, professor of veterinary pathology, and molecular biology and immunology at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies. . . .
In two webcasts held Feb. 29 — one for investors and one for Duchenne muscular dystrophy (DMD) patients and families — PTC Therapeutics addressed the Refuse to File letter it received last week from the U.S. Food and Drug Administration (FDA) stating that PTC’s New Drug Application (NDA) for Translarna (ataluren) to treat DMD was not sufficient . . .
Researchers are looking for people with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial, sponsored by Biogen and Ionis Pharmaceuticals, to test the experimental drug BIIB067 (Ionis-SOD1Rx). BIIB067 is an antisense oligonucleotide, composed of short segments of synthetic genetic material (nucleic acid) that bind to RNA. The drug is designed to . . .
