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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
FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D
On Jan. 2, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation for MYO-102, an investigational gene therapy for limb-girdle muscular dystrophy type 2D (LGMD2D), also known as…
FDA Announces the Approval of TIGLUTIK, an Oral Suspension Form of riluzole, for the Treatment of ALS
On September 6th, the U.S. Food and Drug Administration (FDA) announced the approval of TIGLUTIK, an oral suspension form of riluzole for the treatment of amyotrophic lateral sclerosis (ALS). TIGLUTIK,…
Tags: Drug Approval
From Graduation Onward: MDA National Ambassador Justin Moy Looks Ahead
Spring 2018 has been an exceptionally busy time for National Ambassador Justin Moy. After being named 2018 MDA National Ambassador in March, Justin traveled to Japan with members of his…
Sparking the Live Unlimited Spirit
MDA families experience live unlimited moments every day. Many of them start at MDA Summer Camp.
Tags: Features
FDA Approves Radicava to Treat ALS
Today, on the first Friday of ALS Awareness Month, the U.S. Food and Drug Administration (FDA) approved edaravone (brand name Radicava), to treat ALS (amyotrophic lateral sclerosis). Under development by…
Tags: Drug Approval