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A Tale of Two Siblings: Twin MDA Volunteers Forge Lifelong Connections β and Careers
Twin brother and sister Michael and Amy Schleicherβs story starts where a lot of kidsβ 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
OnΒ 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 Pharmaβs 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 Pharmaβs 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
News on LEMS Treatment and Research
A Q&A with researcher Stephen Meriney, Ph.D.
Tags: Drug Approval, Drug Development, Grants, Research
Making the Workplace Accessible
Think outside the cubicle to find solutions to common accessibility challenges in the office
Tags: Employment
Prepare Yourself
In the face of the everchanging medical landscape, it is important that you prepare yourself and family members to go to the emergency room alone or without the option of…
Tags: Featured Content, Healthcare
The Independent Mindset
When neuromuscular disease impacts your ability to perform daily tasks, cultivating problem-solving skills and support can help you live independently in your home
Tags: College, Featured Content, MDA Resource Center, Personal Care Attendants, Resources, Young Adults
Clinical Trial Alert: Researchers at Neurogene Seek Participants for a CMT4J Natural History Study
Researchers at Neurogene areΒ seeking participantsΒ living withΒ Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able…