Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2–4 years with Rett syndrome
The study was supported by Acadia Pharmaceuticals (San Diego, CA). This study was registered at ClinicalTrials.gov (NCT04988867).
Authors
- Amitha Ananth (ORCID: https://orcid.org/0000-0002-9671-2883)
- Colleen Buhrfiend
- Elizabeth Berry‐Kravis (ORCID: https://orcid.org/0000-0001-7099-1522)
- Amy M Barrett (ORCID: https://orcid.org/0000-0001-8671-8803)
- Jeffrey L. Neul (ORCID: https://orcid.org/0000-0002-5628-5872)
- Timothy Feyma (ORCID: https://orcid.org/0000-0003-0736-8185)
- Tim A. Benke (ORCID: https://orcid.org/0000-0002-6969-5061)
- Eric D. Marsh (ORCID: https://orcid.org/0000-0003-3264-0902)
- David N. Lieberman (ORCID: https://orcid.org/0000-0003-0554-2696)
- Kathie M. Bishop
- Alan K. Percy (ORCID: https://orcid.org/0000-0002-9873-5472)
- Dilesh Doshi (ORCID: https://orcid.org/0009-0006-0970-2158)
- Mona Darwish
- Robin Ryther
- James M. Youakim (ORCID: https://orcid.org/0000-0001-5551-8956)
- Cary Fu (ORCID: https://orcid.org/0000-0001-7720-9249)
- Di An (ORCID: https://orcid.org/0000-0001-6171-7791)
Institutions
- Acadia Pharmaceuticals (United States) (US)
- Rush University Medical Center (US)
- Boston Children's Hospital (US)
- Children's Hospital of Philadelphia (US)
- Washington University in St. Louis (US)
- John F. Kennedy Center for the Performing Arts (US)
- University of Alabama at Birmingham (US)
- Children's Hospital Colorado (US)
- Gillette Children's Specialty Healthcare (US)
- Rush Children's Hospital (US)
- RTI Health Solutions (US)
- Rush University (US)
- Vanderbilt University Medical Center (US)
- University of Colorado Denver (US)
Publication Details
- Journal
- Med
- Published
- 2025-03-04
- DOI
- https://doi.org/10.1016/j.medj.2025.100608
- Citations
- 8
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 14.54
Funders
- ACADIA Pharmaceuticals