Article

Top Caregiver Concerns in Rett syndrome and related disorders: data from the US Natural History Study

2023-03-20

Abstract excerpt

<title>Abstract</title> <p><bold>Objective:</bold> Recent advances in the understanding of neurodevelopmental disorders such as Rett syndrome (RTT) has enabled development of novel therapeutic approaches that are currently undergoing clinical evaluation or are proposed to move into clinical development. Clinical trial success depends on outcome measures that assess clinical features that are most impactful for af...

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Literature Corpus work: dab12339-3ffd-5522-b4d7-b7ff4e755bd3
DOI: 10.21203/rs.3.rs-2566253/v1

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