Claudio Hetz, a professor in the Faculty of Medicine at the University of Chile, was awarded an MDA research grant totaling $294,000 over a period of three years to study a potential new therapy aimed at rescuing motor neurons in amyotrophic lateral sclerosis (ALS). In ALS, some proteins do not fold into their required shape . . .
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 . . .
Christine Vande Velde, associate professor at the University of Montreal CHUM Research Center in Montreal, Quebec (Canada), was awarded an MDA research grant totaling $300,000 over a period of three years to study what goes wrong in amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene. In ALS patients with . . .
When Kathy Payette was in eighth grade, she started experiencing unexplained muscle weakness. Over a period of three years in the mid-1970s, she visited several doctors, all of whom were puzzled by her symptoms. When she was 17, Kathy spent a week at the Mayo Clinic with a team of doctors who concluded she had . . .
Asim Beg, assistant professor at University of Michigan in Ann Arbor, was awarded an MDA research grant totaling $300,000 over a period of three years to study the role of a protein, EphA4, in amyotrophic lateral sclerosis (ALS). High levels of EphA4 correlate with rapid disease progression in ALS patients. Beg and colleagues will work . . .
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 . . .