Using the Bayley-4 and Vineland-3 in Angelman syndrome: barriers, solutions, and challenging items
Clinical trials often rely on the Bayley-4 and Vineland-3 assessments as outcome measures, yet our identified barriers threaten their validity. The associated solutions provide a path forward for improving administration of the Bayley-4 and Vineland-3 in clinical practice, research, and future trials focused on individuals with AS and other intellectual and developmental disabilities.
Authors
- Anjali Sadhwani (ORCID: https://orcid.org/0000-0003-0938-0591)
- Sean N. Halpin (ORCID: https://orcid.org/0000-0001-5624-6083)
- Katherine C. Okoniewski (ORCID: https://orcid.org/0000-0002-5423-1790)
- Anne Wheeler (ORCID: https://orcid.org/0000-0003-2693-5616)
- Angela Gwaltney (ORCID: https://orcid.org/0000-0002-2305-7187)
- Sarah Nelson Potter (ORCID: https://orcid.org/0000-0002-8640-8992)
Institutions
- Boston Children's Hospital (US)
- RTI International (US)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-06-03
- DOI
- https://doi.org/10.1186/s13023-025-03817-x
- Citations
- 2
- Primary Topic
- Genetic Syndromes and Imprinting
- Type
- article
- Field-Weighted Citation Impact
- 3.63
Funders
- Angelman Syndrome Foundation
- Foundation for Angelman Syndrome Therapeutics
- Ultragenyx Pharmaceutical