Safety and efficacy of trofinetide in Rett syndrome: a systematic review and meta-analysis of randomized controlled trials
Abstract Introduction Rett syndrome is a rare genetic neurodevelopmental disorder that predominantly impacts females. It presents with loss of acquired skills, impaired communication, and stereotypic hand movements. Given the limited treatment options for Rett syndrome, there is a dire need for effe...
Main Authors: | , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2024-03-01
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Series: | BMC Pediatrics |
Subjects: | |
Online Access: | https://doi.org/10.1186/s12887-024-04526-3 |