Tokatly Latzer, Itay http://orcid.org/0000-0003-3132-1379
Roullet, Jean-Baptiste http://orcid.org/0000-0001-7403-3620
Afshar-Saber, Wardiya http://orcid.org/0000-0001-9488-7893
Lee, Henry H. C. http://orcid.org/0000-0003-4975-7330
Bertoldi, Mariarita http://orcid.org/0000-0002-2337-9928
McGinty, Gabrielle E.
DiBacco, Melissa L. http://orcid.org/0000-0002-8046-8740
Arning, Erland http://orcid.org/0000-0002-0934-0808
Tsuboyama, Melissa http://orcid.org/0000-0003-4147-7983
Rotenberg, Alexander http://orcid.org/0000-0002-6495-5928
Opladen, Thomas
Jeltsch, Kathrin
García-Cazorla, Àngels
Juliá-Palacios, Natalia
Gibson, K. Michael
Sahin, Mustafa http://orcid.org/0000-0001-7044-2953
Pearl, Phillip L. http://orcid.org/0000-0002-6373-1068
Funding for this research was provided by:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (1R01HD091142)
Article History
Received: 14 September 2023
Accepted: 9 April 2024
First Online: 24 April 2024
Declarations
:
: The SSADHD NHS is approved by the Boston Children’s Hospital Institutional Review Board (#P00029917).
: Not applicable.
: The authors AR and HHCL are co-founders and have equity in Galibra Neuroscience, Inc., which develops treatments for SSADHD including gene replacement therapy discussed in this study.