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The natural history of type 2 Gaucher disease in the 21st century: A retrospective study

OBJECTIVE: To gather natural history data to better understand the changing course of type 2 Gaucher disease (GD2) in order to guide future interventional protocols. METHODS: A structured interview was conducted with parents of living or deceased patients with GD2. Retrospective information obtained...

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Publicado en:Neurology
Autores principales: Roshan Lal, Tamanna, Seehra, Gurpreet K., Steward, Alta M., Poffenberger, Chelsie N., Ryan, Emory, Tayebi, Nahid, Lopez, Grisel, Sidransky, Ellen
Formato: Artigo
Lenguaje:Inglês
Publicado: Lippincott Williams & Wilkins 2020
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7713752/
https://ncbi.nlm.nih.gov/pubmed/32764102
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000010605
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