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Search for: Press Enter to Begin Your Search Skip to content Muscular Dystrophy Association News Research Advocacy Living with Muscular Dystrophy Kids Young Adults Adults MDA Ambassadors Fundraising & Events Fill the Boot Galas Golf Tournaments Lock-Up Muscle Walk Partners Team Momentum Shamrocks Summer Camp MDA.org News Research Advocacy Living with Muscular Dystrophy Kids Young Adults Adults MDA Ambassadors Fundraising & Events Fill the Boot Galas Golf Tournaments Lock-Up Muscle Walk Partners Team Momentum Shamrocks Summer Camp MDA.org Give Strongly An expression of the live unlimited spirit, stories and voices of the MDA community News Clinical Trial Alert: Phase 3 Study of ION363 in Individuals with ALS with Fused in Sarcoma Mutations (FUS-ALS) MDA Staff 05/14/2021 05/14/2021 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, . . . Read More News The Search for ALS Treatments Continues MDA Staff 05/12/2021 05/12/2021 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 . . . Read More Personal Stories Help and Hope for Friedreich’s Ataxia Claire Sykes 05/06/2021 05/14/2021 Bella, a 15-year-old in Fort Worth, Texas, loves jellyfish, horses, anime, Billie Eilish, reading sci-fi and fantasy, and making art. But living with Friedreich’s ataxia (FA) makes it hard for her to engage in some of her favorite activities. “I can draw and paint for only a few minutes before my hand starts to hurt, so I . . . Read More News Research Study Alert: Survey for Individuals with DMD — The Duchenne Muscular Dystrophy Health Index (DMD-HI) MDA Staff 04/12/2021 04/12/2021 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 . . . Read More Personal Stories Introducing New MDA National Ambassador Keisha Greaves and Checking in With Ambassador Ethan LyBrand MDA Staff 04/05/2021 04/12/2021 Beginning in the early 1950s, when public awareness and understanding of muscular dystrophy and related neuromuscular diseases were extremely limited, MDA began calling upon individuals living with these diseases to serve as National Ambassadors, telling their personal stories and inspiring support of MDA. More than 40 MDA National Ambassadors, including children and adults, have met U.S. . . . Read More Kids Early Warning Signs Help Spot Neuromuscular Disease in Children Shaila Wunderlich 04/02/2021 04/21/2021 Every year, Deborah and Jeff Corbett went to their son Josh’s annual checkup with a set of questions. They checked on Josh’s chronic ear troubles and surgeries, inquired about his lagging motor skills, and at his 4-year visit, they asked about his peculiar running gait. When he was 5, they mentioned Josh had difficulty getting . . . Read More News Clinical Trial Alert: Phase 2 Study of Pegcetacoplan in Adults with Sporadic ALS (MERIDIAN) MDA Staff 03/31/2021 03/31/2021 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. . . . Read More News Research Study Alert: Observational Study of a Smartphone Application to Track Progression of ALS and Related Motor Neuron Diseases MDA Staff 03/23/2021 03/30/2021 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 . . . Read More News Clinical Trial Alert: Phase 1/2 Study of AT845 in Late-Onset Pompe Disease MDA Staff 03/16/2021 03/25/2021 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 . . . Read More News Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS MDA Staff 03/10/2021 03/25/2021 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 . . . Read More 1 2 3 4 ... Next Last Need more inspiration? Sign up for MDA news & updates. First Name Last Name Email Connect with MDA Muscular Dystrophy Association National Office 161 N. 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