Pokrzywinski, Robin M.
Goodwin, Bridgett
Dellon, Evan S.
Kodroff, Ellyn
Brooks, Anne
Bailey, Adam
Williams, James
Desai, Nirav K.
Funding for this research was provided by:
This study was funded by Shire Human Genetic Therapies Inc., a member of the Takeda Group of companies
Medical writing support was provided by Iona MacKillop, MSc, Natasha Molle, MSc, and Tsvetana Stoilova, PhD, of PharmaGenesis London, London, UK, and funded by Takeda Pharmaceuticals USA, Inc.
Article History
Received: 10 January 2023
Accepted: 10 October 2023
First Online: 31 October 2023
Declarations
:
: This study protocol was approved by the Ethical and Independent Review Services of the institutional review board, written informed assent was obtained for participants, and written informed consent was obtained for all caregivers of participants.
: Not applicable.
: Robin M. Pokrzywinski and Adam Bailey are employees of Evidera and received funding from Shire Human Genetic Therapies, Inc., a Takeda company, to conduct this study. Bridgett Goodwin, James Williams and Nirav K. Desai are employees of Takeda Development Center Americas, Inc., and are stockholders of Takeda Pharmaceutical Company Limited. Evan S. Dellon has received research funding from Adare Pharmaceuticals/Ellodi Pharmaceuticals, Allakos, Arena Pharmaceuticals/Pfizer, AstraZeneca, Celgene/Receptos/Bristol Myers Squibb, GlaxoSmithKline, Meritage Pharma, Inc., Miraca Life Sciences, Nutricia, Regeneron Pharmaceuticals, Revolo Biotherapeutics and Shire, a Takeda company, and is a consultant for Abbott Laboratories, AbbVie, Adare Pharmaceuticals/Ellodi Pharmaceuticals, Aimmune Therapeutics, Akeso Biopharma, Alfasigma Global, ALK, Allakos, Amgen, Aquilon Pharma, Arena Pharmaceuticals/Pfizer, Aslan Pharmaceuticals, AstraZeneca, Avir Pharma, Biorasi, Calypso Biotech, Celgene/Receptos/Bristol Myers Squibb, Celldex Therapeutics, Inc., EsoCap Biotech, EuPRAXIA, Ferring Pharmaceuticals, GlaxoSmithKline, Gossamer Bio, Holoclara, Inc., Invea, Knightpoint, Landos Biopharma, Lilly, Lucid Diagnostics, Morphic Therapeutic, Nexstone Immunology, Nutricia, Parexel/Calyx Clinical Research Solutions, Phathom Pharmaceuticals, Regeneron Pharmaceuticals, Revolo Biotherapeutics, Robarts Clinical Trials, Inc./Alimentiv, Inc., Salix Pharmaceuticals, Sanofi, Shire, a Takeda company, Target RWE and Upstream Bio, and has received educational grants from Allakos, Holoclara and Invea Therapeutics. Ellyn Kodroff has nothing to disclose. Anne Brooks was an employee of Evidera at the time of the study, and received funding from Shire Human Genetic Therapies, Inc., a Takeda company, to conduct this study.