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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2022 Clinical & Scientific Conference March 15 Highlights

Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care.  Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .

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2022 Clinical & Scientific Conference March 14 Highlights

Today was the first full day of sessions of MDA’s 2022 Clinical & Scientific Conference. For the first time since the start of the COVID-19 pandemic, clinicians, scientists, policymakers, nonprofit, and industry leaders are convening, primarily in-person, for a dynamic and informative five days in Nashville, TN. With more than 975 in-person and 490 virtual . . .

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Reflecting on our 2021 Advocacy Accomplishments

2021 was another challenging year for many, but once again, MDA’s grassroots advocates were relentless in ensuring lawmakers heard their voices. Throughout the year, our volunteers emailed, called, and met with important decision-makers urging them to act on vital public policy that will improve the lives of people living with neuromuscular disease and their families.  . . .

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Muscular Dystrophy Association Awards 18 Grants Totaling Over $1.6 Million for Neuromuscular Disease Research

On Oct. 28, 2021 the Muscular Dystrophy Association (MDA) announced the awarding of 18 new grants totaling over $1.6 million toward neuromuscular disease (NMD) research. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. The newly . . .

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Clinical Trial Alert: Phase 3b Study of Oral Edaravone in Adults with ALS

Researchers at Mitsubishi Tanabe Pharma Development America, Inc. (MTPA) are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in a phase 3b clinical trial to evaluate the safety and efficacy of oral edaravone to treat ALS. Edaravone (brand name Radicava) was designed to help protect cells from damage caused by free radicals, and was . . .

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Clinical Trial Alert: Phase 3 Study of ION363 in Individuals with ALS with Fused in Sarcoma Mutations (FUS-ALS)

Researchers at IONIS Pharmaceuticals are seeking individuals living with amyotrophic lateral sclerosis (ALS) with fused in sarcoma mutations (FUS-ALS) to participate in a phase 3 clinical trial to determine the efficacy of the investigational drug ION363. Drug efficacy will be determined by measuring clinical functioning and survival in trial participants.   The study consists of two parts, . . .

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The Search for ALS Treatments Continues

May is Amyotrophic Lateral Sclerosis (ALS) Awareness Month, and while this past year was different — given the global pandemic we all endured — the research and support for finding new treatments never stopped.  ALS is a neurodegenerative disease of the motor neurons that eventually causes muscular atrophy, paralysis, and death. The cause of ALS is understood . . .

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