Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular Atrophy
Author:
Publisher
Elsevier BV
Subject
Neurology (clinical),General Medicine,Pediatrics, Perinatology and Child Health
Reference27 articles.
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3. The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins;Liu;Cell,1997
4. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?;Burghes;Nat. Rev. Neurosci.,2009
5. Correlation between SMA type and SMN2 copy number revisited: an analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases;Calucho;Neuromuscul. Disord.,2018
Cited by 3 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Gene-Based Therapy for the Treatment of Spinal Muscular Atrophy Types 1 and 2 : A Systematic Review and Meta-Analysis;2023-12-07
2. Spinal muscular atrophy – the effectiveness of treatment and new therapeutic possibilities for selected groups of patients in Poland;Prospects in Pharmaceutical Sciences;2023-06-14
3. Real World Evidence on the Effectiveness of Nusinersen within the National Program to Treat Spinal Muscular Atrophy in Poland;Healthcare;2023-05-22
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