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MDA 2025: Donavon Decker honored for decades of advocacy
Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact in Research, recognizing his efforts across decades on behalf of the muscular dystrophy community. Decker, who has limb-girdle muscular dystrophy (LGMD) type 2D, was honored at the Muscular Dystrophy Association (MDA)’s annual meeting, recently held in Dallas and virtually. “It’s always…
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REGENXBIO Shares Positive Biomarker Data from AFFINITY DUCHENNE Trial of RGX-202 Gene Therapy
REGENXBIO Inc. has reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product delivering a micro-dystrophin transgene via AAV8 for individuals with Duchenne. According to REGENXBIO, the new data from the age 1-3 cohort builds on the favorable safety…
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Mine has been a life of music and joy — and gazing at the stars
Each of my writings in this column has focused on living life with limb-girdle muscular dystrophy. While the mission of Bionews, the parent company of this website, includes sharing what it’s like to live with a chronic progressive condition, I believe it’s also paramount to share other aspects of my existence that make me who…
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MDA 2025: DYNE-101 improves finger function in DM1 clinical trial
Treatment with DYNE-101 is leading to improvements in muscle function — with notable gains in finger function — among people with myotonic dystrophy type 1 (DM1) in a Phase 1/2 clinical trial, according to updated interim data. DM1 patients given the experimental therapy in the ongoing ACHIEVE study (NCT05481879) were able to open and close…
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Essential Gear for Traveling with a Disability
Travel should be exciting, not stressful. However, when you have a disability, packing the right mobility equipment and adaptive travel gear can mean the difference between a smooth adventure and unexpected challenges. Whether you’re heading out for a weekend getaway or an extended trip, having the right equipment can help you feel confident, comfortable, and…
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Restoring heart function in Myotonic Dystrophy Type 1
Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function.
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My 3-year-old is realizing her older brothers with DMD are different
“Mommy, hold me. Mommy, hold me.” These were the words I heard repeatedly from my 3-year-old daughter, Callie, during the four days we spent in the neuromuscular clinic with her older brothers last week. It wasn’t normal for her to want to be held. I’ve never traveled with a stroller for her because she prefers…
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MDA 2025: DMD gene therapy RGX-202 well tolerated, data show
RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing clinical trial, with no serious side effects reported. Interim data from the Phase 1/2 part of the trial, which is sponsored by RGX-202’s developer Regenxbio, also indicate that RGX-202 appears to be working as intended, robustly…
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Insights by Ira: A Celebration of the Best of MDA Center Care Physicians
Prior to the start of 2025, I began the usual and customary exercise of establishing professional and personal goals for the new year. While I have many goals this year, including writing my first fiction novel, expanding my culinary abilities and sharing my love of cooking through online videos, and continuing to have a consistent…
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Sarepta reports death of DMD patient given gene therapy Elevidys
A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the therapy’s developer, Sarepta Therapeutics, said it is continuing to gather and analyze information about this event, which has been reported to relevant health authorities. Elevidys clinical study…
