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January 2023 brings many exciting announcements from CRD, don't miss these updates!
READ MOREIn collaboration with reimbursement expert Eric Faulkner, former global VP of patient value and access at Novartis Gene Therapies, and the Muscular Dystrophy Association, we have developed an initial report to establish a novel pathway to advance treatments for ultra-rare diseases.
This is the first comprehensive approach mapped to date in the U.S. that offers a potential pathway for ultra-rare disease patients to access potentially life-saving therapies. The proposed pathway offers a precedent upon which we can rapidly build and execute this pathway in the U.S.
Complete the form to download the full text of the initial report.
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In this discussion with Dr. Noah Weisleder, PhD, from the Weisleder Lab at Ohio State University College of Medicine, and Rich Horgan, founder and CEO of Cure Rare Disease, we’ll delve into the promising research being conducted by Dr. Weisleders’s lab to develop MyoTRIM - a non-viral, protein-based therapy. We’ll discuss findings from the early-stage research, applications for the treatment of various forms of muscular dystrophy, and more.
Follow the link below to join our patient registry. By joining the registry, you help power our research to develop treatments for ultra-rare disease.
Cure Rare Disease, a non-profit organization (Tax ID number 82-2473513), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.
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