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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
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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
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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
