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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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Embracing a new chapter of life as my longtime caregiver bids farewell
Tomorrow evening, my longtime caregiver, Glenda, will leave her job after 18 years and 10 […]
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Duchenne Secures $12.5 Million in CDMRP Funding for FY25: A Hard-Fought Victory in a Challenging Year
We are humbled and grateful to share that Duchenne muscular dystrophy has been recommended by […]
