Gene therapy denied for Afghan refugee whose father aided U.S.
Orchard Therapeutics' Libmeldy is on the market in Europe, but Medicaid refused to cover the therapy because it's not approved in the U.S.
Source: statnews.com
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Talking rare diseases with the U of M | PressReleasePoint
Each Center for Excellence was selected by NORD in a competitive application process requiring evidence of experience and expertise across multiple specialties to meet the needs of rare disease patients and a history of significant contributions to rare disease patient education, physician training and research.
Source: pressreleasepoint.com
