Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://www.jci.org/articles/view/121658/files/pdf
Reference62 articles.
1. Zwei frühinfantile hereditäre Fälle von progressiver Muskelatrophie unter dem Bilde der Dystrophie, aber anf neurotischer Grundlage
2. Identification and characterization of a spinal muscular atrophy-determining gene
3. Correlation between severity and SMN protein level in spinal muscular atrophy
4. Intragenic telSMN Mutations: Frequency, Distribution, Evidence of a Founder Effect, and Modification of the Spinal Muscular Atrophy Phenotype by cenSMN Copy Number
5. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
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