Systematic Literature Review to Assess the Cost and Resource Use Associated with Spinal Muscular Atrophy Management
Author:
Funder
F. Hoffmann-La Roche
Publisher
Springer Science and Business Media LLC
Subject
Public Health, Environmental and Occupational Health,Health Policy,Pharmacology
Link
https://link.springer.com/content/pdf/10.1007/s40273-021-01105-7.pdf
Reference118 articles.
1. Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy: a literature review. Orphanet J Rare Dis. 2017;12(1):124. https://doi.org/10.1186/s13023-017-0671-8.
2. Wirth B. An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA). Hum Mutat. 2000;15(3):228–37. https://doi.org/10.1002/(SICI)1098-1004(200003)15:3%3c228::AID-HUMU3%3e3.0.CO;2-9.
3. Kolb SJ, Kissel JT. Spinal muscular atrophy. Neurol Clin. 2015;33(4):831–46. https://doi.org/10.1016/j.ncl.2015.07.004.
4. Lorson CL, Hahnen E, Androphy EJ, Wirth B. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci USA. 1999;96(11):6307–11. https://doi.org/10.1073/pnas.96.11.6307.
5. Crawford TO, Paushkin SV, Kobayashi DT, Forrest SJ, Joyce CL, Finkel RS, et al. Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study. PLoS ONE. 2012;7(4): e33572. https://doi.org/10.1371/journal.pone.0033572.
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