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Blog Post | News, Science + Research

MDA Releases New DMD Fact Sheet During Duchenne Muscular Dystrophy Awareness Week

Feb. 13-18, as established by MDA’s colleagues at Parent Project Muscular Dystrophy (PPMD), is Duchenne Muscular Dystrophy Awareness Week, when we recognize both the individuals and families impacted by Duchenne…

Tags: Resources


Blog Post | News, Science + Research

Long-Term Results of Extension Study Validate Surgery as a Treatment for Myasthenia Gravis

Results of an extension study following an MDA-supported clinical trial showed that surgical removal of the thymus in patients with myasthenia gravis (MG) offered benefits for as long as five…

Tags: Clinical Trials, Research


Blog Post | News

Trexo Robotics Releases Trexo Home Walking Aid for Children

Joining a growing number of companies developing robotic devices for those living with mobility impairments, Ontario’s Trexo Robotics just launched the newest in its line of robotic walking aids specifically…

Tags: Innovation, Technology


Blog Post | News, Science + Research

Solid Biosciences Announces Preliminary Results of DMD Gene Therapy Trial

On Feb. 7, Cambridge, Mass.-based Solid Biosciences announced preliminary results from its phase 1/2 clinical trial, called IGNITE DMD, which is designed to assess the safety and efficacy of its lead…

Tags: Clinical Trials, Drug Development, Gene Therapy, Research


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