Archives
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 are seeking participants living with 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…
Sarepta Therapeutics Announces Positive Interim Results in Gene Therapy Trial for LGMD2E and Acquisition of Myonexus Therapeutics
On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle…
Buy a Shamrock, Grow Hope
It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide…
Tags: Fundraising, Shamrocks, Summer Camp
Wisconsin Sen. Tammy Baldwin Introduces Air Carrier Access Amendments Act
Last week, Sen. Tammy Baldwin of Wisconsin introduced the Air Carrier Access Amendments Act (S. 669). MDA commends Sen. Baldwin for championing this legislation that sets forth important protections for…
Clinical Trial Alert: ReveraGen BioPharma Seeks Participants for a Phase 2b DMD Study
Researchers at ReveraGen BioPharma are looking for ambulatory boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 2b study. The goal of the study is to evaluate the…
FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease
On Feb. 25, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to AT-GAA, Amicus’ investigational combination drug therapy ATB200/AT2221 for treating late-onset Pompe disease. The…