Category: News

News

Research Study Alert: Survey for Individuals with DMD — The Duchenne Muscular Dystrophy Health Index (DMD-HI)

MDA Staff

Researchers at University of Rochester are seeking individuals living with Duchenne muscular dystrophy (DMD) to participate in a survey to help with development and validation of a disease-specific, patient-reported outcome measure for clinical trials involving people with DMD. Participants will need to complete an online survey and a follow-up phone interview. The survey will take approximately 20 . . .

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News

Clinical Trial Alert: Phase 2 Study of Pegcetacoplan in Adults with Sporadic ALS (MERIDIAN)

MDA Staff

Researchers at Apellis Pharmaceuticals are seeking individuals living with sporadic amyotrophic lateral sclerosis (ALS) to participate in a two-year phase 2 trial (MERIDIAN) to determine the efficacy of the investigational drug pegcetacoplan. Pegcetacoplan is designed to reduce the activity of the complement system (a component of the immune system), and potentially slow the progression of ALS. . . .

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News

Research Study Alert: Observational Study of a Smartphone Application to Track Progression of ALS and Related Motor Neuron Diseases

MDA Staff

Researchers at Johns Hopkins University School of Medicine are seeking individuals living with amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), or a related motor neuron disease to participate in a study/survey to determine if disease progression can be tracked by a smartphone application. The study uses REDCap, a secure website for medical research, to collect information . . .

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News

Clinical Trial Alert: Phase 1/2 Study of AT845 in Late-Onset Pompe Disease

MDA Staff

Researchers at Audentes Therapeutics, an Astellas company, are seeking individuals living with late-onset Pompe disease (LOPD) to participate in a phase 1/2 study to confirm safety and efficacy of the investigational drug AT845. This gene-replacement therapy may offer the benefit of long-term improvement of motor and respiratory function and quality of life in adults living with . . .

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News

Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS

MDA Staff

Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve as quantifiable, biological, non-clinical measures of Radicava (edaravone) effects on ALS. Observational measures might include assessment of vitals; blood and urine collection; safety assessments; use . . .

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News

Clinical Trial Alert: Phase 3 Study of Zilucoplan in Individuals With Generalized Myasthenia Gravis

MDA Staff

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .

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News

FDA Approves Sarepta Therapeutics’ Amondys 45 for Treatment of DMD Amenable to Skipping Exon 45

Sujatha Gurunathan

On Feb. 25, the US Food and Drug Administration (FDA) granted accelerated approval to casimersen (Amondys 45) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. It is the fourth exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous . . .

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News

Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis

MDA Staff

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .

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News

MDA Welcomes Dr. Don Wood as its New President and CEO

Nancy Intrator

Steve Jobs said, “The only way to do great work is to love what you do.” And MDA’s new president and CEO, Donald S. Wood, PhD, surely loves what he does. “The mission of this organization has been a lifelong passion for me,” Dr. Wood says. After receiving his doctorate in Physiology from Washington State . . .

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Adults

What You Need to Know About Open Enrollment in 2020

Mark Fisher

Open enrollment — the period each year when people are eligible to purchase new insurance or make changes to their existing insurance plans — is upon us. Buying health insurance can be confusing, but it’s critically important that everyone in the neuromuscular community have coverage. We are pleased to share some FAQs below to help . . .

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