Drug treatment for spinal muscular atrophy type I

BACKGROUND: Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor neuron 1 (SMN1) gene on chromosome 5, or a heterozygous deletion in combination with a point mutation in the second SMN1 allele. This results in degeneration of anterior horn cells, which leads to prog...

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Dades bibliogràfiques
Publicat a:Cochrane Database Syst Rev
Autors principals: Wadman, Renske I, van der Pol, W Ludo, Bosboom, Wendy MJ, Asselman, Fay‐Lynn, van den Berg, Leonard H, Iannaccone, Susan T, Vrancken, Alexander FJE
Format: Artigo
Idioma:Inglês
Publicat: John Wiley & Sons, Ltd 2019
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6905354/
https://ncbi.nlm.nih.gov/pubmed/31825542
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD006281.pub5
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