Archives
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…
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
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…
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…
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