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

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

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

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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MDA Advocacy’s 2020 Year in Review

There’s no doubt 2020 has been a challenging year, but the commitment and passion of our MDA advocates have truly been a bright spot. As we close out the year, let’s recap your amazing accomplishments. Access to care and therapies from day one Thanks to the actions of advocates, together we made sure the lifesaving . . .

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Amylyx Announces More Results from Study of AMX0035 to Treat ALS

On Oct. 16, Amylyx announced an update from its CENTAUR trial evaluating the effects of the investigational drug AMX0035 (a combination of the small molecules sodium phenylbutyrate and taurursodiol) on overall survival in people with amyotrophic lateral sclerosis (ALS). The current study followed each participant in the CENTAUR trial for up to three years from . . .

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Biogen Announces Positive Results from Phase 1/2 Study of Tofersen to Treat a Genetic Form of ALS

On July 8, Biogen announced positive results from its phase 1/2 clinical trial of the investigational therapy tofersen (BIIB067) for treatment of amyotrophic lateral sclerosis (ALS) caused by mutation of the superoxide dismutase 1 gene (SOD1). The primary endpoints of the study — assessment of safety, tolerability, and drug dynamics in the body — demonstrated . . .

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What the COVID-19 Pandemic Can Teach Us About the Isolation Those Living With Neuromuscular Disease Experience Every Day

In March 2020, MDA asked our advocates how the novel coronavirus pandemic was impacting their lives. New Yorker Robert Paulson shared his story — and the universal truth about how neuromuscular disease (NMD), like COVID-19, creates isolation. But for those living with amyotrophic lateral sclerosis (ALS) and other forms of NMD, that isolation doesn’t always . . .

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