Title : The effect of hydroxyurea in spinal muscular atrophy cells and patients.

Pub. Date : 2008 May 15

PMID : 18166199






2 Functional Relationships(s)
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1 The effect of HU on SMN2 gene expression and clinical manifestations was evaluated. Hydroxyurea survival of motor neuron 2, centromeric Homo sapiens
2 CONCLUSIONS: Treating with HU enhanced SMN2 gene expression in SMA cells and showed slight trend towards improvement in some clinical outcome measures in SMA patients which suggests HU may be safe to use in SMA patients but larger randomized, placebo-controlled, double-blind trials are needed to further investigate its efficacy. Hydroxyurea survival of motor neuron 2, centromeric Homo sapiens