Challenges and opportunities in spinal muscular atrophy therapeutics
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Elsevier BV
Reference118 articles.
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1. Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study;Frontiers in Neurology;2024-07-22
2. Analysis of blood concentrations and clinical application of risdiplam in patients with spinal muscular atrophy using ultra‐high performance liquid chromatography–tandem mass spectrometry;Biomedical Chromatography;2024-07-15
3. Breaking genetic shackles: The advance of base editing in genetic disorder treatment;Frontiers in Pharmacology;2024-03-06
4. Deciphering spinal muscular atrophy: the need for more research;The Lancet Neurology;2024-02
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