Villar-Pazos, Sabrina
Thomas, Laurel
Yang, Yunhan http://orcid.org/0000-0001-6000-7210
Chen, Kun
Lyles, Jenea B.
Deitch, Bradley J.
Ochaba, Joseph
Ling, Karen http://orcid.org/0000-0002-5895-6684
Powers, Berit
Gingras, Sebastien http://orcid.org/0000-0003-2607-8642
Kordasiewicz, Holly B.
Grubisha, Melanie J.
Huang, Yanhua H. http://orcid.org/0000-0003-1770-0196
Thomas, Gary http://orcid.org/0000-0003-1976-7183
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (NS123649)
U.S. Department of Health & Human Services | NIH | National Institute of Diabetes and Digestive and Kidney Diseases (DK112844, DK114855)
U.S. Department of Health & Human Services | NIH | National Institute of Diabetes and Digestive and Kidney Diseases
NORD (National Organization for Rare Disorders) PACS1 Syndrome Research Foundation
Article History
Received: 26 January 2023
Accepted: 29 September 2023
First Online: 17 October 2023
Competing interests
: BP, HBK, JO, and KL are employees and shareholders of Ionis Pharmaceuticals. The other authors declare no competing interests.