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I’m taking steps that will lead me away from caregiver burnout
I wrote last week that I have been overwhelmed this summer by the demands of being a mom and caregiver. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (DMD). Caring for…
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Highlights from PPMD’s 2026 Annual Conference
Whether you joined us in Orlando or are catching up from home, the learning opportunities from PPMD’s 2026 Annual Conference continue. We’re excited to share that recordings and resources from PPMD’s 2026 Annual Conference are now available! From the latest updates in Duchenne and Becker research to practical care conversations, educational sessions, and inspiring community…
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New milestones hit in MD trial testing treatment to turn off faulty gene
Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed to turn on or off a specific gene without changing the underlying DNA — for facioscapulohumeral muscular dystrophy (FSHD), a genetic disease that characteristically affects the muscles of the face, shoulders, and upper arms. Epicrispr…
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I almost lost my garden to Becker MD. One tool brought it back.
There’s a certain rhythm to gardening that I’ve always loved. The early mornings, the quiet work of turning soil, the satisfaction of seeing something grow because you cared for it. In my backyard, I’ve built raised beds filled with vegetables and herbs — nothing fancy, but enough to keep my hands busy and my mind…
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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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Satellos reports positive interim data from adults treated with SAT-3247
CureDuchenne was an early funder of Satellos, and we are pleased to share that they have reported 6-month interim data from 4 individuals (aged 21-28) treated with SAT-3247. These individuals showed reduced muscle fat fraction, an increase in upper limb effort, increased handgrip strength, and reductions in creatine kinase. See press release here: https://ir.satellos.com/news/news-details/2026/Satellos-Reports-Six-Month-Interim-TRAILHEAD-Data-Showing-Reduced-Muscle-Fat-Fraction-Increased-Effort-Stable-Strength-Lower-CK-and-Favorable-Safety-Profile-in-DMD-Adults-Treated-with-SAT-3247/default.aspx The…
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A Purple Parade profile amplifies my disability advocacy
Last month, the Purple Parade featured my story on its website. Reading my profile prompted me to reflect on why I continue speaking up about living with Duchenne muscular dystrophy (DMD) and what I hope these conversations might achieve. The Purple Parade is Singapore’s largest disability-inclusion movement, bringing people with disabilities, their families, businesses, community…
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Italfarmaco reports positive data from the EPIDYS trial and its ongoing open-label extension of givinostat in Duchenne
Italfarmaco reported new data from the Phase 3 EPIDYS trial, showing that quantitative MRI measures of muscle mass and fat fraction were significantly different between the treatment and placebo groups. Interim analyses from the open-label extension study also indicate a delay in loss of ambulation (LoA), with median age of LoA =17.33 years with treatment,…
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Annual campaign raises $641K to send youths to MDA Summer Camp
This year’s Be Their Muscle campaign has raised more than $641,000 to support MDA Summer Camp for children and adolescents with muscular dystrophy and other neuromuscular diseases, and to help advance research and care for these patient populations. Resulting from a partnership between the Muscular Dystrophy Association (MDA) and Burn Boot Camp, this year’s campaign has included 400…
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Preparing for the Deramiocel FDA Advisory Committee Meeting: How to Submit Written Testimony
The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held virtually, and a live stream will be available for public viewing. The Duchenne community is actively working together to prepare for this important meeting. Following the announcement,…
