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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Veröffentlicht in:Orphanet J Rare Dis
Hauptverfasser: Lusakowska, Anna, Jedrzejowska, Maria, Kaminska, Anna, Janiszewska, Katarzyna, Grochowski, Przemysław, Zimowski, Janusz, Sierdzinski, Janusz, Kostera-Pruszczyk, Anna
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2021
Schlagworte:
Research
Online Zugang: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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