Pharmacotherapy for Spinal Muscular Atrophy in Babies and Children: A Review of Approved and Experimental Therapies
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Pediatrics, Perinatology and Child Health
Link
https://link.springer.com/content/pdf/10.1007/s40272-022-00529-8.pdf
Reference125 articles.
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3. Sugarman EA, Nagan N, Zhu H, Akmaev VR, Zhou Z, Rohlfs EM, et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. 2012;20(1):27–32.
4. Vill K, Kölbel H, Schwartz O, Blaschek A, Olgemöller B, Harms E, et al. One year of newborn screening for SMA—results of a German Pilot Project. J Neuromuscul Dis. 2019;6(4):503–15.
5. Kay DM, Stevens CF, Parker A, Saavedra-Matiz CA, Sack V, Chung WK, et al. Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy. Genet Med. 2020;22(8):1296–302.
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