Consensus on Gene Replacement Therapy for the Treatment for Spinal Muscular Atrophy
Author:
Publisher
National Medical Research Center for Childrens Health
Subject
Ocean Engineering
Reference30 articles.
1. Verhaart I.E.C., Robertson A., Wilson I.J., Aartsma-Rus A., Cameron S., Jones C.C., 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. Calucho M., Bernal S., Alías L., March F., Venceslá A., Rodríguez-Álvarez F.J., et al. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases. Neuromuscul. Disord. 2018; 28(3): 208–15. https://doi.org/10.1016/j.nmd.2018.01.003
3. Russian clinical guidelines «Proximal spinal muscular atrophy». Available at: https://cr.minzdrav.gov.ru/schema/593_3 (in Russian)
4. Saffari A. Novel challenges in spinal muscular atrophy – how to screen and whom to treat? Ann. Clin. Transl. Neurol. 2019; 6(1): 197–205. https://doi.org/10.1002/acn3.689
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