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At 45, I’m aware of the many turns along my Duchenne MD path
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
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Satellos asks FDA to clear Phase 2 trial of DMD treatment
Satellos Bioscience asked the U.S. Food and Drug Administration (FDA) for clearance to conduct a Phase 2 clinical trial testing SAT-3247 in children with Duchenne muscular dystrophy (DMD) who are able to walk. The three-month study will randomly assign participants to SAT-324, taken as a pill, or a placebo. Its goal is to assess the…
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Simply Stated: Updates in Facioscapulohumeral muscular dystrophy (FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic disorder that may affect all muscles across the lifespan of an individual. While FSHD has historically been detected in muscles in the face (facio), shoulders (scapulo), and upper arms (humeral), new data derived from AI analysis of whole-body MRI scans challenges the historical view that FSHD is limited…
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The physical challenges of DMD haven’t dimmed my love of sports
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
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My late brother was my greatest teacher, in life and Duchenne
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
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Izzy Camilleri Merges High Fashion and Adaptive Clothing Design
Toronto-based fashion designer Izzy Camilleri knows high fashion. Celebrated in the fashion world for decades, Izzy has dressed stars such as David Bowie, Meryl Streep, Angelina Jolie, and Daniel Radcliffe, and her work has been featured in magazines like Vogue and InStyle. But it wasn’t until she received a unique design request in 2004 that…
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LGMD gene therapy gets FDA orphan drug designation
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy type 2i/R9 (LGMDR9). The FDA gives this designation to experimental therapies designed to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. The designation confers incentives to companies…
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Capricor plans Deramiocel resubmission to the FDA, pursuing both cardiac and skeletal muscle indications in Duchenne
Capricor Therapeutics, which received early funding from CureDuchenne, provided an update on their Deramiocel program in Duchenne after their recent FDA meeting. This meeting with the FDA was to address the concerns in the Complete Response Letter (CRL) that Capricor received from the FDA in July 2025, and align on a path forward toward consideration…
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MDA Ambassador Guest Blog: Life Lessons on My Journey with Becker Muscular Dystrophy
Jon Bruns is 56 years old. He is originally from South Dakota and now lives in Ham Lake, Minnesota. Jon was diagnosed with Beckers muscular dystrophy (BMD) in his early 20’s. He and his wife of over 22 years have one daughter, who is a college student. Jon is an accountant and finance professional and…
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Catch Up on PPMD Together: Charlotte
This past weekend, PPMD held its last in person PPMD Together event of 2025 in Charlotte, North Carolina. Over two days, families, individuals with Duchenne and Becker muscular dystrophy, clinicians, researchers, and industry partners gathered to connect, build community, and learn together. Throughout the event, attendees were able to share their stories and experiences with…
