Variation in cognitive function over time in Gaucher disease type 3

OBJECTIVE: To identify relevant efficacy parameters essential in designing clinical trials for brain-penetrant therapies for Gaucher disease, we evaluated cognitive function longitudinally in 34 patients with Gaucher disease type 3 seen at the NIH Clinical Center. METHODS: Individuals were tested wi...

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Pubblicato in:Neurology
Autori principali: Steward, Alta M., Wiggs, Edythe, Lindstrom, Taylor, Ukwuani, Somto, Ryan, Emory, Tayebi, Nahid, Roshan Lal, Tamanna, Lopez, Grisel, Schiffmann, Raphael, Sidransky, Ellen
Natura: Artigo
Lingua:Inglês
Pubblicazione: Lippincott Williams & Wilkins 2019
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6937490/
https://ncbi.nlm.nih.gov/pubmed/31719137
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000008618
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