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Observation of the natural course of type 3 spinal muscular atrophy: data from the polish registry of spinal muscular atrophy
BACKGROUND: Spinal muscular atrophy (SMA) is one of the most frequent and severe genetic diseases leading to premature death or severe motor disability. New therapies have been developed in recent years that change the natural history of the disease. The aim of this study is to describe patients inc...
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| Pubblicato in: | Orphanet J Rare Dis |
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| Autori principali: | , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
BioMed Central
2021
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7992780/ https://ncbi.nlm.nih.gov/pubmed/33761963 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01771-y |
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