PMC Links for Books (Select 5112792) - PMC

Result Filters

Links from Books

Items: 3

Select item 101529801.

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

Amy G. Feldman, Julie A. Parsons, Cullen M. Dutmer, Aravindhan Veerapandiyan, Einar Hafberg, Nolan Maloney, Cara L. Mack

J Pediatr. Author manuscript; available in PMC 2023 May 2.

Published in final edited form as: J Pediatr. 2020 Oct; 225: 252–258.e1. Published online 2020 May 28. doi: 10.1016/j.jpeds.2020.05.044

PMCID:: PMC10152980

Abstract Article PDF–2.0M

Select item 66522812.

Onasemnogene Abeparvovec for Spinal Muscular Atrophy: The Costlier Drug Ever

Rajiv Mahajan

Int J Appl Basic Med Res. 2019 Jul-Sep; 9(3): 127–128. doi: 10.4103/ijabmr.IJABMR_190_19

PMCID:: PMC6652281

Article PDF–370K

Select item 57767123.

Natural History of Infantile-Onset Spinal Muscular Atrophy

Stephen J. Kolb, Christopher S. Coffey, Jon W. Yankey, Kristin Krosschell, W. David Arnold, Seward B. Rutkove, Kathryn J. Swoboda, Sandra P. Reyna, Ai Sakonju, Basil T. Darras, Richard Shell, Nancy Kuntz, Diana Castro, Julie Parsons, Anne M. Connolly, Claudia A. Chiriboga, Craig McDonald, W. Bryan Burnette, Klaus Werner, Mathula Thangarajh, Perry B. Shieh, Erika Finanger, Merit E. Cudkowicz, Michelle M. McGovern, D. Elizabeth McNeil, Richard Finkel, Susan T. Iannaccone, Edward Kaye, Allison Kingsley, Samantha R. Renusch, Vicki L. McGovern, Xueqian Wang, Phillip G. Zaworski, Thomas W. Prior, Arthur H.M. Burghes, Amy Bartlett, John T. Kissel

Ann Neurol. Author manuscript; available in PMC 2018 Dec 8.

Published in final edited form as: Ann Neurol. 2017 Dec; 82(6): 883–891. Published online 2017 Dec 8. doi: 10.1002/ana.25101