Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

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 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 . . .

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Education in Your Community

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 . . .

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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 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). . . .

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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 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, . . .

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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 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 . . .

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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 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 . . .

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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 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, . . .

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Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study

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 . . .

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In Their Shoes: 65 Years of IAFF’s Fight for MDA

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 . . .

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