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Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy
Spinal muscular atrophy (SMA) is a neurodegenerative disease primarily characterized by a loss of spinal motor neurons, leading to progressive paralysis and premature death in the most severe cases. SMA is caused by homozygous deletion of the survival motor neuron 1 (SMN1) gene, leading to low level...
Gorde:
| Argitaratua izan da: | Drugs |
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| Egile Nagusiak: | , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
Springer International Publishing
2018
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5829132/ https://ncbi.nlm.nih.gov/pubmed/29380287 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-018-0868-8 |
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