Clinical Trial Alert: Phase 3 Study of investigational drug in Adults Living with ALS

Researchers at Cytokinetics, Inc. are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in the phase 3 COURAGE-ALS clinical trial to evaluate the safety and effectiveness of the investigational drug. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost, causing the skeletal muscles they control to become weak and nonfunctional. . . .

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Brain-Computer Interface Technology Could Improve Life for Millions

It’s been half a century since University of California, Los Angeles, computer science professor Jacques Vidal coined the term “brain-computer interface” (BCI) and produced the first peer-reviewed papers on the idea of interpreting brain signals with a computer. Today, BCI offers huge potential to improve the lives of millions of people around the world, including . . .

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Research Study Alert: Study of Wearable Gait Sensor in People with or without Neuromuscular Disease

Researchers at the University of California, Davis Neuromuscular Research Lab are seeking individuals with or without neuromuscular disease (ages 2 years or older) to participate in a research study to test the feasibility of using a wearable gait sensor to collect data as part of a new outcome measure for use in future clinical trials.  This . . .

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FDA Approves Alexion’s Ultomiris for Treatment of gMG

On April 28, the US Food and Drug Administration (FDA) granted approval to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United . . .

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Simply Stated: Inclusion Body Myositis, Revisited

Inclusion body myositis (IBM) is a rare, progressive muscle disease characterized by chronic muscle inflammation and weakness. It is estimated that approximately 20,000 people in the United States (US) have IBM, though the exact prevalence is unknown. IBM usually develops after age 50 and is more likely to affect men than women. The disease progresses . . .

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Natural History Trial of BMD Now Enrolling

On April 14, Edgewise Therapeutics announced the initiation of an Edgewise-funded observational trial in individuals with Becker muscular dystrophy (BMD). The goal of trial is to understand the progression of disease in individuals with BMD as assessed by functional measures and imaging endpoints.  BMD is a genetic disease caused by a mutation in the dystrophin . . .

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Promoting Diversity in Rare Disease Research

Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .

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Researchers Announce Results from a Phase 3 Study of Corticosteroid Dosing to Treat DMD

On April 5, an international research team, headed by Michela Guglieri at Newcastle University and Robert Griggs at the University of Rochester Medical Center, announced positive results at the American Academy of Neurology (AAN meeting) in Seattle, WA from the phase 3 FOR DMD study of corticosteroid dosing regimens for treatment of people with Duchenne . . .

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Research Study Alert: Survey for People with Muscular Dystrophy and Their Caregivers about Quality of Life

Researchers at Goldsmiths University of London are seeking people living with muscular dystrophy (MD) and their primary caregivers to participate in a survey about their quality of life. The aim of the survey is to better understand how quality of life of an individual and their primary caregiver is affected by progressing MD. In this study, . . .

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