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Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive genetic disorder resulting in degeneration of α-motor neurons of the anterior horn and proximal muscle weakness. It is the leading cause of genetic mortality in children younger than 2 years. It affects ∼1 in 11,000 live births. In 95% of cases...
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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4142828/ https://ncbi.nlm.nih.gov/pubmed/25147906 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/adt.2014.587 |
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