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National Institute for Health January 2022

Experimental gene therapy reverses sickle cell disease for years

Rare Diseases: Although Limited, Available Evidence Suggests Medical and Other Costs Can Be Substantial

Introductory Chapter: Advances in the Diagnosis and Management of Rare Diseases

Drug Pricing, a Complex Issue Affecting the Rare Disease Community

FAQs About Rare Diseases

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases