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Your Citizen Science At Work
By sharing your anonymous data through The Duchenne Registry, you are strengthening the power of a 15-year-old network of patient-powered data that is used to improve care for people living with Duchenne and increase our understanding of the disorder. Data is shared with researchers, fueling the fight to end Duchenne and helping to speed the…
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Phase 1/2 update ‘encouraging’ for DMD gene therapy RGX-202
The experimental gene therapy RGX-202 has been found to be well tolerated at a high dose, with biomarker data indicating it is working as designed to increase production of the microdystrophin protein in boys with Duchenne muscular dystrophy (DMD). That’s according to updated interim findings from the ongoing Phase 1/2 AFFINITY DUCHENNE clinical trial (NCT05693142),…
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I’m at a crossroads in life, once again
Starting last week, I’ve been trying to get my life back on track after enduring a few difficult months. As I recently shared, life has been challenging since I left my job in April. Then, in May, I faced an unexpected hospitalization, and June brought relationship tension. In July, my mental health issues resurfaced, exacerbating…
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PGN-ED051, skipping exon 51, showing benefits in Phase 2 trial
A low dose of PGN-ED051, PepGen’s investigational exon 51-skipping therapy, safely increased dystrophin protein levels in people with Duchenne muscular dystrophy (DMD), according to early Phase 2 clinical trial data. The therapy’s effects either were comparable to or greater than what has been observed in studies of other exon 51-skipping therapies at a similar dose…
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Clinical Trials & Outcome Measures in Duchenne
Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as well. We need more sensitive outcome measures that are more inclusive to a broad population of individuals living with Duchenne…
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Ladies Luncheon Returns to Austin Country Club on September 26 for an “Afternoon in NYC” Benefiting CureDuchenne
Annual Event Features an Afternoon of Fashion and Fun to Raise Funds to Help Find a Cure for Duchenne Muscular Dystrophy AUSTIN, Texas (August 5, 2024) – CureDuchenne, a leading global nonprofit focused on funding and finding a cure for Duchenne muscular dystrophy, announced the return of the sixth annual Ladies Luncheon at the Austin…
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cTAP Study Indicates Duchenne Muscular Dystrophy Clinical Trial Enrollment Criteria Should Expand Beyond Ambulatory Status
Study Co-Funded by CureDuchenne and cTAP Highlights Need for More Specific Enrollment Criteria That Could Increase Patient Participation and Drive More Comprehensive Therapeutic Evaluation CAMBRIDGE, Mass., July 24, 2024 – The Collaborative Trajectory Analysis Project (cTAP) and international collaborators have announced evidence to support using a more sophisticated set of criteria than is typically used…
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In ‘Muscle Memoirs,’ I’ll share tales of life with LGMD
Muscular dystrophy entered my life in the fall of 1984. I was 11 years old and beginning my sixth year of school. Along with neighborhood friends, I would walk to and from my elementary school every day. That September, I remember feeling very tired on the short walk down the hill, as well as a…
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Finding beauty in how Duchenne has shaped our lives
Duchenne muscular dystrophy (DMD) touches every part of my family’s life. Jason, my husband of 23 years, and I have seven children. Three of them — Max, 18, Rowen, 15, and Charlie, 13 — live with DMD. We’re now in the middle of what could be one of the most exciting periods of our family’s…
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REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202
Link to press release We are thrilled to share the latest data from RegenXBio’s microdystrophin gene therapy, AFFINITY DUCHENNE®. RGX-202 is a one-time gene therapy for Duchenne designed to deliver a novel microdystrophin gene via AAV8. RGX-202 is differentiated from other microdystrophin gene therapies in that it contains a larger portion of the C-Terminal domain,…
