Elivaldogene autotemcel approved for treatment of cerebral adrenoleukodystrophy (CALD) in males: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

Genet Med Open. 2023 Nov 15;1(1):100835. doi: 10.1016/j.gimo.2023.100835. eCollection 2023.

Anna I Scott^{1

2}, Kanwaldeep K Mallhi^{3

4}, Jaya Ganesh⁵, Wei-Liang Chen^{6

7}; ACMG Therapeutics Committee8∗documents@acmg.net

¹ Department of Laboratories, Seattle Children's Hospital, Seattle, WA.
² Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA.
³ Department of Pediatric Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY.
⁴ Department of Pediatrics, State University of New York at Buffalo, Buffalo, NY.
⁵ Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY.
⁶ Department of Genetics, Pediatrics, and Neurology, George Washington University, Washington, DC.
⁷ Center for Neuroscience Research, Children's National Medical Center, Washington, DC.

No abstract available

Keywords: Cerebral; Elivaldogene; Gene therapy; Newborn screening; X-linked adrenoleukodystrophy.