Rett syndrome: The Natural History Study journey

• Main Authors: Alan K. Percy (Author), Timothy A. Benke (Author), Eric D. Marsh (Author), Jeffrey L. Neul (Author)
• Format: Bog
• Udgivet: Wiley, 2024-09-01T00:00:00Z.
• Fag: article
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Abstract Understanding clinical features and disease progression of Rett syndrome (RTT) and establishing clinical trial readiness was enhanced by the RTT Natural History Study (NHS). The NHS benefited from two key developments: one, the Orphan Drug Act passed by Congress in 1983 defining criteria fo...