P241: Real-world use of trofinetide: A survey of dosing strategies from US Rett syndrome centers of excellence
Trofinetide was approved by the US Food and Drug Administration in March 2023 for treating Rett syndrome in patients aged ≥2 years, with twice daily weight-banded dosing. Here, we present real-world examples of alternative dosing strategies.
Authors
- Amitha Ananth (ORCID: https://orcid.org/0000-0002-9671-2883)
- Arthur Beisang (ORCID: https://orcid.org/0000-0002-9095-6174)
- Caroline B. Buchanan
- Christopher W. Beatty (ORCID: https://orcid.org/0000-0002-8352-6694)
- David N. Lieberman (ORCID: https://orcid.org/0000-0003-0554-2696)
- Emily de los Reyes (ORCID: https://orcid.org/0000-0002-1123-6903)
- Alyssa M. Peckham (ORCID: https://orcid.org/0000-0002-9638-7005)
- Alan K. Percy (ORCID: https://orcid.org/0000-0002-9873-5472)
- Robin Ryther
- Bernhard Suter (ORCID: https://orcid.org/0000-0002-0127-7322)
- Louise Cosand
- Grace Correa
Institutions
- Acadia Pharmaceuticals (United States) (US)
- Boston Children's Hospital (US)
- Nationwide Children's Hospital (US)
- Baylor College of Medicine (US)
- Washington University in St. Louis (US)
- University of Alabama at Birmingham (US)
- Gillette Children's Specialty Healthcare (US)
- Greenwood Genetic Center (US)
- Boston Children's Museum (US)
- The Ohio State University (US)
Publication Details
- Journal
- Genetics in Medicine Open
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1016/j.gimo.2025.102206
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00