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A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls
BACKGROUND: Clinical trials to test safety and efficacy of drugs for patients with spinal muscular atrophy (SMA) are currently underway. Biomarkers that document treatment-induced effects are needed because disease progression in childhood forms of SMA is slow and clinical outcome measures may lack...
Zapisane w:
| Wydane w: | PLoS One |
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| Główni autorzy: | , , , , , , , , , , , |
| Format: | Artigo |
| Język: | Inglês |
| Wydane: |
Public Library of Science
2016
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| Hasła przedmiotowe: | |
| Dostęp online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5125671/ https://ncbi.nlm.nih.gov/pubmed/27893852 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0167087 |
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