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 intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .
At MDA, we believe in the power of community and the importance of building relationships with families going through similar experiences. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage educational events taking place across the country. MDA Engage Community Education Seminars are one-day events . . .
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 to determine clinically meaningful outcome measures for use in future clinical trials. This five-year study will consist of 10 visits (one visit every six months). . . .
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 muscular dystrophy type 2E (LGMD2E), also known as beta-sarcoglycanopathy. The first three patients dosed demonstrated significant expression of the protein beta-sarcoglycan in the muscle fiber, . . .
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 to raise a little green (more than $323 million, to be exact) both to send kids to MDA Summer Camp and to fund groundbreaking research . . .
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 travelers with disabilities. Even though the Air Carrier Access Act, which prohibits discrimination based on disability, was passed more than 30 years ago, individuals with . . .
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 safety and efficacy of vamorolone. Researchers hope this therapy may improve muscle strength and endurance with a more favorable safety profile compared to the current . . .
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 company’s combination drug therapy pairs ATB200, a synthetic human acid alpha-glucosidase enzyme meant to replace the missing enzyme in Pompe disease, with pharmacological chaperone AT2221, . . .
Researchers at Amicus Therapeutics are looking for adults with late-onset Pompe disease (LOPD) to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be randomized to . . .
In 1952 in South Boston, Charles Crowley was desperate for help. His two sons were living with Duchenne muscular dystrophy (DMD) and needed care. The family needed resources. Charles called a good friend, George Graney. George did what good friends do for friends who need help: He called on his friends, too. Specifically, George asked . . .