Rarely Speaking: Living Well with MD – Confirmation
The post Rarely Speaking: Living Well with MD – Confirmation appeared first on Muscular Dystrophy News .
Muscular Dystrophy News – The Web's Daily Resource for Muscular Dystrophy News
The Web's Daily Resource for Muscular Dystrophy News
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A busy week ends with a baby shower, a stomach bug, and a lesson learned
The week before school started was pure chaos. We had only been home from vacation long enough to unpack and start the laundry before we had to go to open houses on Monday at two of the kids’ schools, and then close on our new house. If that weren’t hectic enough, before the week was […] The post A busy week ends with a baby shower, a stomach bug, and a lesson learned appeared first on Muscular…
Biotech raises $90M for late-stage testing of FSHD treatment
Epicrispr Biotechnologies has raised $90 million in financing to support late-stage clinical testing of EPI-321, the company’s epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). “This financing marks a pivotal milestone for Epicrispr as we advance EPI-321 and the next generation of programmable epigenetic medicines,” Amber Salzman, PhD, CEO of Epicrispr, said in a…
Safety is a key component of accessibility in public transportation
Last year, our family’s wheelchair-accessible van broke down due to a fault in its hydraulic ramp. From March to May 2025, my caregiver and I had no choice but to take the bus to my office. It was the first time I’d used public transportation in a decade. Back then, I didn’t need a ventilator […] The post Safety is a key component of accessibility in public transportation appeared first on…
Rarely Speaking: Living Well with MD: Real-World Hacks & Daily Realities
The post Rarely Speaking: Living Well with MD: Real-World Hacks & Daily Realities appeared first on Muscular Dystrophy News .
Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy fights every single battle necessary to end Duchenne and Becker. We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge therapies, and a community of support. We invest deeply in therapeutic options for this generation of Duchenne and Becker patients and in research that will benefit future generations. Our…
FDA OKs expansion of DMD stem cell trial, allows 30 more boys
The U.S. Food and Drug Administration (FDA) has allowed the enrollment of up to 30 additional boys with Duchenne muscular dystrophy (DMD) in a clinical study testing an experimental stem cell therapy. The Phase 2 trial (NCT06579352) testing the treatment from Signature Biologics has enrolled five boys, ages 5 to 10, at two sites in […] The post FDA OKs expansion of DMD stem cell trial, allows 30…
A vacation with close friends was a perfect way to end the summer
My family recently spent a week in Indiana with friends at a cabin on a lake, and it was so good for us. Our two families met more than a decade ago when our sons were all little boys, and we have remained friends throughout our separate journeys with Duchenne muscular dystrophy (DMD). My husband […] The post A vacation with close friends was a perfect way to end the summer appeared first on…
US developer gearing up for first-in-human trial of Duchenne cell therapy
A U.S. biopharmaceutical company is gearing up to launch its first clinical trial of a novel cell therapy designed to restore lost muscle cells in people with Duchenne muscular dystrophy (DMD). Texas-based IPS Heart announced that it is preparing an investigational new drug application or IND — a formal request to the U.S. Food and Drug […] The post US developer gearing up for first-in-human trial…
New issues arise while seeking answers to my progressing symptoms
My regular readers know I’ve been losing function in my right arm, making many activities of daily living challenging. The pain, weakness, and stiffness I experience when I try to do anything with that arm began suddenly and has progressed rapidly this year. I’m now experiencing similar symptoms in my left arm. Given that rapid […] The post New issues arise while seeking answers to my progressing…
Leaky muscle fibers may help drive damage in muscular dystrophy
When muscle fibers fail to properly seal tiny tears in their membrane, as occurs in some forms of muscular dystrophy, the resulting leakage can change the surrounding environment and attract immune cells that contribute to further muscle damage, a mouse study suggests. The findings suggest that the tissue surrounding muscle fibers may play an active […] The post Leaky muscle fibers may help drive…
I help people through a neuromuscular diagnosis and whatever comes next
“I don’t even know what question to ask.” I’ve heard some version of that sentence hundreds of times. Sometimes it’s from a parent whose child was just diagnosed with Duchenne muscular dystrophy. Sometimes it’s a husband whose wife has been diagnosed with ALS. Other times it’s someone living with spinal muscular atrophy, Charcot-Marie-Tooth disease, limb-girdle […] The post I help people through a…
How my attitude toward health checkups has changed over the years
Shortly after being diagnosed with muscular dystrophy in 1985, I returned to the hospital for a checkup and to review my treatment plan. I thought the doctors might tell me to eat an apple, take some aspirin, and rest up. I was 12 years old, so what did I know? In my mind, I’d go […] The post How my attitude toward health checkups has changed over the years appeared first on Muscular Dystrophy…
Expanded access program to bring DMD treatment to patients in 1 US state
The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients with Duchenne muscular dystrophy (DMD). Del-zota, which is being developed by Avidity Biosciences, is specifically designed to treat DMD in people with disease-causing mutations that are amenable to…