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Hogan Family
Raising Awareness and Funding Duchenne Research: Christian Hogan and his 22-year-old son Micah, who has Duchenne, envisioned this event after attending a reggae show in Long Beach several years ago.
Duchenne Muscular Dystrophy Moves Closer to Newborn Screening Recommendation
In a promising development, the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHNDC) has advanced the nomination of Duchenne muscular dystrophy (DMD) for inclusion in the Recommended Uniform Screening Panel (RUSP), a list of disorders that are screened for at birth.
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate Phase 1 Clinical Trial of ENTR-601-44 for the Potential Treatment of Duchenne Muscular Dystrophy
– First participant is expected to be dosed in September 2023 with data anticipated in the second half of 2024 – – Cash runway extended through the end of 2025 […]
Edgewise Therapeutics Announces Positive 12-Month Topline Results From The ARCH Open Label Study Of EDG-5506 In Adults With Becker Muscular Dystrophy (BMD)
The positive results from the 12-month ARCH study support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies, associated with EDG-5506 administration, has the potential to preserve and improve muscle function while preventing disease progression in dystrophinopathies. Observations from ARCH identified key factors, including the optimal dosing strategy of EDG-5506, for the design of a potentially registrational trial. A pivotal cohort, GRAND CANYON, has been added to the CANYON study and is anticipated to begin enrollment in the third quarter of 2023.
FDA Grants Accelerated Approval to Sarepta Therapeutics’ SRP-9001 (ELEVIDYS) Gene Therapy for Duchenne Muscular Dystrophy
SEE UPDATE FROM OCTOBER 30, 2023 HERE CLICK BELOW FOR A FEW WORDS FROM OUR TEAM AND SAREPTA: Sarepta Therapeutics’ gene therapy for Duchenne, SRP-9001, has been granted Accelerated Approval […]
Locanabio Announces Equity Investment from CureDuchenne Ventures to Support Development of snRNA Exon Skipping Approach for Duchenne Muscular Dystrophy (DMD)
“CureDuchenne is a leader in funding pioneering research aimed at advancing the best possible therapies for DMD patients, and we value their vote of confidence and financial support of our snRNA-mediated exon skipping platform,” said Jim Burns, Ph.D., Locanabio’s chief executive officer.
Debra Miller Representing Patient Advocacy Community
My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.
Why Mothers Should Be Celebrated Every Day
There is no way to be a normal parent, when you have abnormal circumstances. The important thing is that you keep getting up every day loving those kids. You may think it’s not enough, but your children see you differently than you see yourself. I have days that I feel like super mom and days that I feel like a huge failure. “It’s just too hard,” I mumble to myself as I crawl into bed at night unsure of how I will possibly do the whole thing again tomorrow.
CureDuchenne Ventures invests in Insmed Incorporated to support the development of a next-generation gene therapy with targeted intrathecal delivery and the potential to produce a larger dystrophin construct.
CureDuchenne Ventures announces an investment in Insmed Inc. to support the development of INS1201, their next generation intracerebroventricular (ICV) delivered AAV to directly address limitations of current gene therapy approaches.