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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…
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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…
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ASK ME ANYTHING: Endocrine & Bone Health for People DBMD
ASK ME ANYTHING: Endocrine & Bone Health for People with DBMD Dr. David Weber, Pediatric Endocrinologist at Children’s Hospital of Philadelphia (CHOP) Recorded: Wednesday, August 5, 2026 Watch HERE The post ASK ME ANYTHING: Endocrine & Bone Health for People DBMD appeared first on CureDuchenne.
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ASK ME ANYTHING: Navigating IEPs and 504 Plans
Watch our webinar (in English and Spanish) with helpful insights on who is eligible, common school supports, transition planning, and how to build a strong partnership with your child’s school. Recorded: August 2026Watch HERE The post ASK ME ANYTHING: Navigating IEPs and 504 Plans appeared first on CureDuchenne.
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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…
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In Case You Missed It…
Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
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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…
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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 exon…
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MDA’s Guide to the Fair Housing Act
The Civil Rights Movement of the 1960s prompted the passage of several landmark civil rights laws, including the Fair Housing Act (FHA), which was enacted in 1968 to protect people from discrimination when renting, buying, or financing a home. What is the FHA? “From a broad perspective, the FHA is about preventing discriminatory treatment for…
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FDA advisers reject evidence for Duchenne heart therapy
An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the effectiveness of the experimental cell therapy deramiocel for treating heart muscle disease in people with Duchenne muscular dystrophy (DMD). In a company press release, Capricor Therapeutics, the therapy’s developer, said the question before the committee…
