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Blog Post | Advocacy, News, Science + Research

Arkansas to Screen Babies for SMA

This week, Arkansas Gov. Asa Hutchinson signed into law a bill, HB 1074, that requires the state to add spinal muscular atrophy (SMA) to its newborn screening panel. SMA is…

Tags: Advocacy Updates, Newborn Screening


Blog Post | Get Involved, Personal Stories

Planned Giving: A Different Kind of Donation

On Dec. 23, 2018, the MDA office of Minnesota and the Dakotas received its biggest gift ever — and it came as a surprise from one man who felt connected…

Tags: Fundraising


Blog Post | News, Science + Research

Catalyst Pharmaceuticals Announces Positive Results from Phase 2b Trial of Firdapse in Patients with Muscle-Specific Kinase Antibody Positive Myasthenia Gravis (MuSK-MG)

Italian researchers published positive results in the journal SAGE Open Medicine of a Phase 2b trial of Firdapse.

Tags: Clinical Trials, Drug Approval, Drug Development, Research


Blog Post | News, Science + Research

FDA Awards Fast Track Status to RNS60, Revalesio’s Investigational Therapy for ALS

On Jan. 4, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to Tacoma, Wash.-based Revalesio Corporation’s experimental drug for amyotrophic lateral sclerosis (ALS), RNS60. Fast Track status…

Tags: Clinical Trials, Drug Development, Research


Blog Post | News, Science + Research

Clinical Trial Alert: Brainstorm Cell Therapeutics Seeks Participants for a Phase 3 ALS Study

Researchers at Brainstorm Cell Therapeutics are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a Phase 3 study designed to help researchers evaluate the effects of mesenchymal…

Tags: Clinical Trials, Drug Development, Research


Blog Post | Advocacy, Get Involved, News, Science + Research

Newborn Screening for DMD and BMD: MDA Needs Your Help

Early identification and treatment for neuromuscular disorders are essential to optimize health outcomes. Newborn screening, which identifies health issues via a blood test taken soon after birth, is essential to…

Tags: Advocacy Updates, Newborn Screening, Research


Blog Post | Advocacy, News, Science + Research

MDA Awards Venture Philanthropy Grant to AcuraStem to Develop Treatment for ALS

The Muscular Dystrophy Association has awarded an MDA Venture Philanthropy (MVP) grant totaling $300,000 over two years to AcuraStem to support preclinical development of a novel small molecule therapeutic for…

Tags: Grants, Research, Research Advances


Blog Post | News, Science + Research

Clinical Trial Alert: Orphazyme Seeks Participants for a Phase 3 ALS Study

Researchers at Orphazyme are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the ORARIALS-01 Phase 3 study. The study is designed to help researchers evaluate the effects…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development, Research


Blog Post | News, Science + Research

Clinical Trial Alert: Catabasis Seeks Participants for a Phase 3 DMD Study

Researchers at Catabasis are looking for boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study (PolarisDMD). The goal of the study is to evaluate the safety…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development, Research


Blog Post | News, Science + Research

Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

On Jan. 3, Wave Life Sciences announced that its upcoming Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with Duchenne muscular dystrophy (DMD) who are amenable…

Tags: Clinical Trials, Drug Approval, Drug Development, Exon Skipping, Gene Therapy, Research, suvodirsen