Five Questions with Researcher Antoni Barrientos

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 . . .

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$3 Million NIH Grant Propels Vamorolone to Phase 2 Testing for Treatment of DMD

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 . . .

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Workshop Will Update ALS Clinical Trial Guidelines

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 . . .

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Phase 3 Trial for Experimental Drug Tirasemtiv Currently Recruiting Participants with ALS

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 . . .

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Eli Lilly Provides Update on Phase 3 Tadalafil Trial for DMD

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 . . .

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