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Biomarkers in Rare Disorders: The Experience with Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by homozygous mutations of the SMN1 gene. Based on clinical severity, three forms of SMA are recognized (type I–III). All patients have at least one (usually 2–4) copies of a highly homologous gene (SMN2) which pro...

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Библиографические подробности
Главные авторы: Tiziano, Francesco D., Neri, Giovanni, Brahe, Christina
Формат: Artigo
Язык:Inglês
Опубликовано: Molecular Diversity Preservation International (MDPI) 2010
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3039940/
https://ncbi.nlm.nih.gov/pubmed/21339974
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms12010024
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