A cross-sectional and longitudinal evaluation of serum creatinine as a biomarker in spinal muscular atrophy

Abstract Objective Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by proximal muscle weakness and atrophy. The increasing availability of disease-modifying therapies has prompted the development of biomarkers to facilitate clinical assessments. We explore...

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Hlavní autoři:	Xin Zhao, Zhenxiang Gong, Han Luo, Zehui Li, Rong Gao, Kangqin Yang, Wenhua Deng, Sirui Peng, Li Ba, Yang Liu, Min Zhang
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	BMC 2024-12-01
Edice:	Orphanet Journal of Rare Diseases
Témata:	Spinal muscular atrophy Creatinine Biomarkers Nusinersen
On-line přístup:	https://doi.org/10.1186/s13023-024-03515-0
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