Lenmeldy (atidarsagene autotemcel) for individuals with early metachromatic leukodystrophy (MLD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
Genet Med Open. 2025 Jun 24:3:103432.
doi: 10.1016/j.gimo.2025.103432.
eCollection 2025.
1 Division of Medical Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA.
2 qRare International, LLC, Pleasanton, CA.
3 Department of Clinical Genomics and Neurology, Mayo Clinic, Rochester, MN.
4 Division of Clinical Genetics, Department of Pediatrics, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.
5 Division of Pediatric Allergy, Immunology, and Bone Marrow Transplant, Department of Pediatrics, University of California San Francisco, San Francisco, CA.