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Gene discovered that can protect against severe muscle disease
A specific gene may play a key role in new treatments that prevent muscle in […]
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MDA 2024: Gene therapy’s benefits most pronounced in young boys
Among ambulatory boys with Duchenne muscular dystrophy (DMD), a one-time treatment with investigational gene therapy […]
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Here’s What You Missed at the MDA Clinical and Scientific Conference
This year’s Muscular Dystrophy Association Clinical and Scientific Conference was full of presentations on research, […]
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When siblings take a back seat to their brothers with Duchenne MD
I don’t want to miss a thing. Yes, as I typed that, I sang the […]
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MDA 2024: Myotonic dystrophy type 1 reverses with AOC 1001
Long-term treatment with AOC 1001 continues to be safe and may reverse the progression of […]
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REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
RegenXBio shared additional interim data from their Phase 1/2 AAV micro-dystrophin gene therapy clinical trial […]
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REGENXBIO Shares New Positive Initial Efficacy Data From AFFINITY DUCHENNE Trial
REGENXBIO Inc. has shared an update on the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 […]
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PPMD Celebrates 30th Anniversary and Three Decades of Progress with Annual Advocacy Conference
Over the past three decades, PPMD has driven Congress to enact legislation that has transformed federal […]
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PPMD Hosts Town Hall on Clinical Trials During 2024 Advocacy Conference
The PPMD Town Hall on Clinical Trials, taking place on Monday, March 4th during PPMD’s […]
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A blessing in disguise: What has my FSHD given me?
I’m reading “Eyes to the Wind: A Memoir of Love and Death, Hope and Resistance” […]
