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  • Participants Sought for RA101495 MG Trial December 8, 2017
    Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 2 clinical trial, sponsored by Ra Pharmaceuticals, to test the experimental drug RA101495 in people with generalized MG who are positive for acetylcholine receptor autoantibodies. Administered subcutaneously (an injection under the skin), RA101495 is designed to prevent the body’s attack on […]
  • Five Questions with Neuromuscular Disease Researcher James Lupski December 7, 2017
    James Lupski, Cullen Professor of Molecular and Human Genetics and professor of pediatrics at Baylor College of Medicine in Houston, was awarded an MDA research grant totaling $300,000 over three years to facilitate new gene discovery and new biological insights into the pathobiology of neuromuscular disease. Please describe your current research. Determining genetic diagnoses in […]
  • Dyla, Who Lives With SMA, is Gaining Strength Thanks to Spinraza December 6, 2017
    From the moment their daughter was diagnosed with spinal muscular atrophy (SMA) type 2 in 2009, Dyla Gutierrez’s parents have focused on one thing: helping her preserve her strength. “I mean, this is our hope and dream for Dyla. We want Dyla to be stronger. We want her to have every opportunity that every other […]
  • Simply Stated: What’s a Biomarker? December 1, 2017
    A biomarker is any biological indicator that doctors or researchers can objectively measure and evaluate to determine the state of an individual’s health, confirm disease onset and progression, or gauge whether an experimental treatment is working or not. Some common, everyday types of biomarkers include body temperature, blood pressure, pulse, heart rate, and the presence […]
  • Solid Biosciences Launches DMD Gene Therapy Trial November 30, 2017
    Solid Biosciences has announced the launch of its first clinical trial for SGT-001, the company’s experimental gene transfer therapy for Duchenne muscular dystrophy (DMD). The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD. Enrollment at the first trial […]

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