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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 […]
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Capricor announces that individuals with Duchenne treated with deramiocel had significantly less progression in loss of upper limb function
Capricor Therapeutics received early funding from CureDuchenne, and today Capricor released new data reporting that […]
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Dyne announces new long-term data from DMD clinical trial skipping Exon 51
CureDuchenne provided early funding to Dyne Therapeutics, and is pleased to share that Dyne has […]
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Individual with Duchenne dies from acute liver failure after gene therapy treatment
Sarepta Therapeutics has shared that a young man with Duchenne passed away following treatment with […]
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MDA 2025: Deramiocel linked to sustained arm function preservation
Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the […]
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2025 MDA Clinical & Scientific Conference Updates
2025 MDA Conference Updates We’re at the 2025 MDA Clinical & Scientific Conference this week […]
