-
The Hidden Power of Self-Care in Caregiving
Caregivers often give so much of themselves that sometimes their own needs fade into the background. Yet the stories shared here reveal a powerful truth: Caring for yourself is not selfish — it’s essential. Each of the caregivers featured here has learned that strength is built in the pauses — a moment of quiet, a…
-
Meet Our 2025 MDA Quest Photo Contest Winners
Power play Congratulations to our 2025 Quest Media photo contest winner, Paula Saxton, of Doylestown, Pennsylvania, who submitted this photo of her son, Jake. Sports photographer Karla Donohoe captured this moment of celebration after Jake scored a goal in the opening game of the 2025 North American Powerhockey Cup. His team, the Philadelphia Flyers PowerPlay,…
-
FDA Approves New Safety Warning and Revised Indication for ELEVIDYS
Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients treated with the product. This comes after Sarepta shared in July 2025 that the agency requested and the company agreed…
-
Protected: PPMD Continues to Drive Progress with Your Support
This content is password protected. To view it please enter your password below: Password: The post Protected: PPMD Continues to Drive Progress with Your Support appeared first on Parent Project Muscular Dystrophy.
-
Long-term Agamree data show better growth, lower fracture risk
Agamree (vamorolone), a type of corticosteroid approved to help preserve muscle function in people with Duchenne muscular dystrophy (DMD), appears to work as well as traditional corticosteroids at maintaining walking ability, but carries a lower risk of side effects such as broken bones, eye problems, and stunted growth. That’s according to a new analysis announced…
-
MDA Ambassador Guest Blog: How Finding Community Helped Me Face the Fear of Having My Son Tested for CMT
Kevin Crowley is a 48-year-old father of two, husband to the most beautiful woman in the world, dad of a yellow Labrador Retriever, and a coach of the world’s greatest 5th-6th grade youth football team. He was diagnosed with Charcot-Marie-Tooth disease (CMT)1A in his late 20’s. He lives with depression and PTSD and is also…
-
Dancing to the beat and singing to the melody to find a cure for FSHD
I’m a member of the Western Pennsylvania chapter of the FSHD Society, which serves as a support group and a partner in the nationwide Walk & Roll to Cure FSHD campaign aimed at raising funds for the search for a cure for facioscapulohumeral muscular dystrophy (FSHD). The Walk & Roll campaign strives to raise about…
-
Clinical Research Opportunity: Phase 3 Study of Cladribine in People with Generalized Myasthenia Gravis (gMG)
Researchers at EMD Serono Research & Development Institute, Inc. are working to better understand generalized myasthenia gravis (gMG) and study effectiveness of a potentially new treatment. The study People who have generalized myasthenia gravis (gMG) may be eligible to participate in a phase 3 clinical trial to evaluate the safety and efficacy of the investigational therapy…
-
My Journey to Gratitude is Paved with Acceptance and Advocacy
I’m John Krepps and I am living with Charcot Marie Tooth disease, also known as CMT. I live in Pittsburgh Pennsylvania. (so yes, I’m a Steelers fan.) I’m married to my beautiful wife, Tracy, father to a beautiful daughter, Rhiannon, and Pap to our granddaughters Sophia and Maddie. Also, fun facts about me: My wife…
-
Clinical Research Alert: Phase 1 Study of AMX0114 in Adults with ALS
Researchers at Amylyx Pharmaceuticals, Inc. are seeking adults with amyotrophic lateral sclerosis (ALS) to participate in a phase 1 clinical trial (LUMINA) to evaluate the safety and efficacy of the investigational therapy AMX0114 to treat ALS. AMX0114 is designed to reduce the levels of calpain-2, an enzyme linked to the degeneration and death of neurons in…
