Researchers are looking for people with type 1A Charcot-Marie-Tooth disease (CMT1A) to participate in a new pivotal phase 3 clinical trial of the investigational drug PXT3003. The trial aims to determine whether PXT3003 is effective and well-tolerated in people with CMT1A. PXT3003 is an oral low-dose combination of three different drugs designed to target the . . .
Researchers at The Ohio State University are looking for adults, ages 18 years or older, with paramyotonia congenita to participate in a phase 2 open-label study being sponsored by Gilead Science to test the experimental drug ranolazine. Ranolazine currently is approved by the U.S. Food and Drug Administration to treat chest pain in people with . . .
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 . . .
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 . . .
Marathon Pharmaceuticals announced today that it is expanding patient access to deflazacort, its investigational therapy for Duchenne muscular dystrophy (DMD). Medical centers across the country are now participating in the open-label expanded access program (EAP) called ACCESS DMD to provide deflazacort to qualified U.S. patients at no charge. Deflazacort, a corticosteroid, works as an anti-inflammatory . . .
Patients, their families and caregivers, researchers and clinicians are invited to gain insight into the testing process for experimental therapies to treat amyotrophic lateral sclerosis (ALS) via a live webcast this month. The ALS Clinical Trials Guidelines 2016 Workshop, an international effort that seeks to renew and update the current ALS Clinical Trials Guidelines, will . . .
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 . . .
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 . . .
