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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 months of faithfully serving our family here in Singapore. She’ll be returning to the Philippines to be reunited with her husband and three daughters, whom she hasn’t seen regularly for the past eight and a half years. Her departure fills…
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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 House and Senate Defense Appropriations leadership for $12.5 million in federal funding for Fiscal Year 2025 (FY25) through the Congressionally Directed Medical Research Programs (CDMRP) at the Department of Defense (DoD). This achievement reflects the tireless efforts of our community—the…
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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 individuals treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). Capricor reports that this equates…
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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 released new long-term data from the DELIVER trial in individuals amenable to skipping Exon 51. At the 6-month time point, individuals treated with 20 mg/kg of DYNE-251 every 4 weeks had a mean absolute dystrophin expression of 8.72% of normal…
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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 ELEVIDYS, an AAV-delivered micro-dystrophin gene therapy. Sarepta has shared that the individual suffered from acute liver failure after treatment, and will provide more information as they further gather and analyze the data. This disease is unrelenting and new therapeutics are…
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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 U.S., continued to show an ability to preserve upper limb function in boys and men with Duchenne muscular dystrophy (DMD) over the long term. That’s according to analyses of about five years worth of data spanning the Phase 2 HOPE-2…
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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 and are pleased to share research updates from companies presenting on the latest advancements in Duchenne muscular dystrophy treatments. Here are the latest press releases: Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy…
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A Safety Update on ELEVIDYS
We are deeply saddened by the loss of a young member of our community following treatment with ELEVIDYS. According to Sarepta, the individual with Duchenne received ELEVIDYS and later suffered from complications of acute liver failure. This represents the first reported case of acute liver failure resulting in death following treatment with ELEVIDYS. According to…
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Making an Impact on Day 1 of the MDA Clinical and Scientific Conference
“This is a milestone year,” said Donald S. Wood, PhD, MDA’s President and CEO, as he kicked off the 2025 MDA Clinical & Scientific Conference on March 17, in Dallas. He welcomed more than 2,000 neuromuscular researchers, clinicians, allied healthcare providers, industry professionals, and advocacy organizations attending during MDA’s 75th anniversary year. Donald S. Wood,…
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MDA 2025: Conference begins, marking another year of progress
The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science and care related to a range of neuromuscular diseases. This year’s conference will bring together hundreds of participants from 39 countries, Morgan Roth,…
