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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
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
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
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…
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…
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
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.
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
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
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…