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Spinal muscular atrophy
Spinal muscular atrophy (SMA) is a potentially devastating and lethal neuromuscular disease frequently manifesting in infancy and childhood. The discovery of the underlying mutation in the survival of motor neurons 1 (SMN1) gene has accelerated preclinical research, leading to treatment targets and...
Bewaard in:
Gepubliceerd in: | Neurotherapeutics |
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Hoofdauteurs: | , |
Formaat: | Artigo |
Taal: | Inglês |
Gepubliceerd in: |
Springer New York
2008
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Onderwerpen: | |
Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4514700/ https://ncbi.nlm.nih.gov/pubmed/19019300 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nurt.2008.08.007 |
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