Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

We report 2 cases of transient, drug-induced liver failure after this therapy.

Source: The Journal of Pediatrics - May 26, 2020 Category: Pediatrics Authors: Amy G. Feldman, Julie A. Parsons, Cullen M. Dutmer, Aravindhan Veerapandiyan, Einar Hafberg, Nolan Maloney, Cara L. Mack Tags: Brief Reports Source Type: research