International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA
A pipeline of collaborative clinical outcome assessment development and validation research in Rett syndrome can now be designed, aiming to have fit-for-purpose measures that can evaluate meaningful change, to serve future clinical trials and clinical practice.
Authors
- Sarika U. Peters (ORCID: https://orcid.org/0000-0001-7439-5402)
- Amitha Ananth (ORCID: https://orcid.org/0000-0002-9671-2883)
- Frank J. Symons (ORCID: https://orcid.org/0000-0001-9833-4807)
- Breanne J. Byiers (ORCID: https://orcid.org/0000-0002-7645-1479)
- Kathleen J. Motil (ORCID: https://orcid.org/0000-0002-5713-8399)
- Jeffrey L. Neul (ORCID: https://orcid.org/0000-0002-5628-5872)
- Christopher W. Beatty (ORCID: https://orcid.org/0000-0002-8352-6694)
- Tim A. Benke (ORCID: https://orcid.org/0000-0002-6969-5061)
- Gillian S. Townend (ORCID: https://orcid.org/0000-0002-5448-9046)
- Jenny Downs (ORCID: https://orcid.org/0000-0001-7358-9037)
- Dominique C. Pichard (ORCID: https://orcid.org/0000-0002-6492-622X)
- Eric D. Marsh (ORCID: https://orcid.org/0000-0003-3264-0902)
- Paramala Santosh (ORCID: https://orcid.org/0000-0003-4830-5893)
- Helen Leonard (ORCID: https://orcid.org/0000-0001-6405-5834)
- Joseph P. Horrigan (ORCID: https://orcid.org/0000-0002-9336-5820)
- Melissa Raspa (ORCID: https://orcid.org/0000-0003-3791-3367)
- James M. Youakim (ORCID: https://orcid.org/0000-0001-5551-8956)
- Bernhard Suter (ORCID: https://orcid.org/0000-0002-0127-7322)
- Walter E. Kaufmann (ORCID: https://orcid.org/0000-0002-8561-8453)
- Andrew C. Dietz (ORCID: https://orcid.org/0000-0003-2810-2466)
- Al Jacobs
- Nupur Garg
Institutions
- Acadia Pharmaceuticals (United States) (US)
- Boston Children's Hospital (US)
- Albert Einstein College of Medicine (US)
- University of Minnesota (US)
- Nationwide Children's Hospital (US)
- Montefiore Medical Center (US)
- Children's Hospital of Philadelphia (US)
- Emory University (US)
- Duke University (US)
- The Kids Research Institute Australia (AU)
- The University of Western Australia (AU)
- RTI International (US)
- Baylor College of Medicine (US)
- King's College London (GB)
- Curtin University (AU)
- John F. Kennedy Center for the Performing Arts (US)
- University of Alabama at Birmingham (US)
- International Rett syndrome Foundation (US)
- Children's Hospital Colorado (US)
- Rome Foundation (US)
- Maudsley Hospital (GB)
- Children's Nutrition Research Center at Baylor College of Medicine (US)
- The Ohio State University (US)
- University of Reading (GB)
- University of Pennsylvania (US)
- Vanderbilt University Medical Center (US)
- University of Colorado Denver (US)
- Center for Autism and Related Disorders (US)
Publication Details
- Journal
- Trials
- Published
- 2024-12-21
- DOI
- https://doi.org/10.1186/s13063-024-08678-6
- Citations
- 6
- Primary Topic
- Genetics and Neurodevelopmental Disorders
- Type
- article
- Field-Weighted Citation Impact
- 3.30
Funders
- International Rett Syndrome Foundation
- Grace Science Foundation
- Ionis Pharmaceuticals
- GW Pharmaceuticals
- ACADIA Pharmaceuticals
- AveXis
- Marinus Pharmaceuticals
- LouLou Foundation
- International Foundation for CDKL5 Research
- Stan Perron Charitable Foundation
- National Institutes of Health