Personal take on this article: This study explores the potential of Thrombospondin-4 (TSP4) as a cerebrospinal fluid (CSF) biomarker for monitoring therapy response in pediatric patients with Spinal Muscular Atrophy (SMA). SMA is a severe childhood neurodegenerative disease characterized by the loss of motor neurons, leading to muscle weakness and atrophy. Currently, there is a lack of reliable biomarkers that can indicate treatment response in SMA. Researchers conducted proteomic analyses …

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Personal take on this article: This study evaluates the effectiveness of Nusinersen, a drug for treating spinal muscular atrophy (SMA), in adolescents and adults. The researchers conducted a systematic review and meta-analysis of 14 studies involving 539 patients aged 13–72 years, predominantly with SMA Type II or III.  Nusinersen was found to improve or stabilize motor function over 24 months. For the Hammersmith Functional Motor Scale–Expanded (HFMSE), patients showed an …

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