The Use of Real-World Data to Support the Assessment of the Benefit and Risk of a Medicine to Treat Spinal Muscular Atrophy
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Publisher
Springer International Publishing
Link
https://link.springer.com/content/pdf/10.1007/978-3-031-26328-6_20
Reference35 articles.
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2. Lefebvre S, Bürglen L, Reboullet S et al (1995) Identification and characterization of a spinal muscular atrophy-determining gene. Cell 80(1):155–165. https://doi.org/10.1016/0092-8674(95)90460-3.
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4. Mercuri E, Finkel RS, Muntoni F et al (2018) Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul Disord 28(2):103–115. https://doi.org/10.1016/j.nmd.2017.11.005.
5. Munsat TL, Davies KE (1992) International SMA consortium meeting. Neuromuscul Disord 2(5–6):423–8. https://doi.org/10.1016/s0960-8966(06)80015-5.
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