Chen Y, Yang D, Huang X, Feng J, Zhao Q, Huang H
Front Neurol. 2024; 15:1391613.
PMID: 39076847
PMC: 11284043.
DOI: 10.3389/fneur.2024.1391613.
Li D, Yang J, Wang X, Yang L, Luo R, Huang S
Brain Behav. 2024; 14(7):e3630.
PMID: 39034388
PMC: 11260551.
DOI: 10.1002/brb3.3630.
Bzdok J, Czibere L, Burggraf S, Landt O, Maier E, Roschinger W
PLoS One. 2024; 19(6):e0306329.
PMID: 38941330
PMC: 11213327.
DOI: 10.1371/journal.pone.0306329.
Vill K, Tacke M, Konig A, Baumann M, Baumgartner M, Steinbach M
J Neurol. 2024; 271(5):2787-2797.
PMID: 38409538
PMC: 11055798.
DOI: 10.1007/s00415-024-12188-5.
Yang Y, Yuan P, Li M, Jiang L, Hong S
Zhongguo Dang Dai Er Ke Za Zhi. 2021; 23(10):1038-1043.
PMID: 34719420
PMC: 8549636.
DOI: 10.7499/j.issn.1008-8830.2106025.
Quality of Life Outcomes According to Differential Nusinersen Exposure in Pediatric Spinal Muscular Atrophy.
Weaver M, Yuroff A, Sund S, Hetzel S, Halanski M
Children (Basel). 2021; 8(7).
PMID: 34356583
PMC: 8305818.
DOI: 10.3390/children8070604.
New Therapeutics Options for Pediatric Neuromuscular Disorders.
Flotats-Bastardas M, Hahn A
Front Pediatr. 2020; 8:583877.
PMID: 33330280
PMC: 7719776.
DOI: 10.3389/fped.2020.583877.
Advances in Treatment of Spinal Muscular Atrophy - New Phenotypes, New Challenges, New Implications for Care.
Schorling D, Pechmann A, Kirschner J
J Neuromuscul Dis. 2019; 7(1):1-13.
PMID: 31707373
PMC: 7029319.
DOI: 10.3233/JND-190424.
Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients.
Lochmuller H, Evans D, Farwell W, Finkel R, Goemans N, de Lemus M
J Neuromuscul Dis. 2018; 5(2):131-133.
PMID: 29865093
PMC: 6004907.
DOI: 10.3233/JND-180325.
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany.
Pechmann A, Langer T, Schorling D, Stein S, Vogt S, Schara U
J Neuromuscul Dis. 2018; 5(2):135-143.
PMID: 29689734
PMC: 6004898.
DOI: 10.3233/JND-180315.
Treatment Advances in Spinal Muscular Atrophy.
Bharucha-Goebel D, Kaufmann P
Curr Neurol Neurosci Rep. 2017; 17(11):91.
PMID: 28983837
PMC: 5678931.
DOI: 10.1007/s11910-017-0798-y.