Archives
Clinical Trial Alert: PTC Therapeutics Seeks Participants for a Phase 3 DMD Study
Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study. The goal of the study is to characterize the long-term…
Introducing New MDA National Ambassador Tana Zwart, and Checking in With Ambassadors Faith Fortenberry and Justin Moy
Since the early 1950s, not long after the Muscular Dystrophy Association’s formation, America’s young people living with muscular dystrophy and related neuromuscular diseases have stepped forward to share their stories,…
Tags: Ambassadors, Community, Summer Camp, Young Adults
MDA Partners with the Broad Institute on Rare Genomes Project for Limb-Girdle Muscular Dystrophy
The Muscular Dystrophy Association (MDA) is pleased to announce a collaboration with the Broad Institute of MIT and Harvard, which aims to bring genetic diagnoses to more individuals living with…
Tags: Genetic Testing, Grants, Research
Muscular Dystrophy Association Bridges Clinical and Academic Neuromuscular Research for the First Time at Annual Meeting
With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a…
Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1
On April 8, Audentes Therapeutics announced that it will expand its gene-targeted therapy platform to include developing experimental drugs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).…
A Tale of Two Siblings: Twin MDA Volunteers Forge Lifelong Connections — and Careers
Twin brother and sister Michael and Amy Schleicher’s story starts where a lot of kids’ stories do. They looked up to their big brother, Matt, and when he did something…
Muscular Dystrophy Association Awards Eight Grants Totaling More Than $2 Million for ALS Research
Today, the Muscular Dystrophy Association (MDA) announced the awarding of eight new grants totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which…
Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping
On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular…
FDA Grants Orphan Drug Designation to Acceleron Pharma’s ACE-083 Muscle Growth Drug for Charcot-Marie-Tooth Disease
On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by…
Tags: Clinical Trials, Drug Development, Research
What Happened to You
A young man with SMA answers this question by becoming a student leader
Tags: College, From Where I Sit, Young Adults