Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1 | Zendy

Beth Solomon | Zendy; Andrew C. Smith | Zendy; Ninet Sinaii | Zendy; Nicole Y. Farhat | Zendy; Monique C. King | Zendy; Leonza Machielse | Zendy; Forbes D. Porter | Zendy

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Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1

Author(s) -

Beth Solomon,

Andrew C. Smith,

Ninet Sinaii,

Nicole Y. Farhat,

Monique C. King,

Leonza Machielse,

Forbes D. Porter

Publication year - 2020

Publication title -

jama neurology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 5.298

H-Index - 231

eISSN - 2168-6157

pISSN - 2168-6149

DOI - 10.1001/jamaneurol.2020.3241

Subject(s) - medicine , swallowing , interquartile range , dysphagia , cohort , population , pediatrics , cohort study , surgery , environmental health

Niemann-Pick disease, type C1 (NPC1) is a progressive neurovisceral disease with no US Food and Drug Administration-approved therapy. Miglustat, a drug used off-label in the United States for the treatment of NPC1, appears to stabilize neurologic disease progression. Several prospective trials suggest that miglustat stabilizes oropharyngeal swallowing function; however, its effect on dysphagia and aspiration risk has not been demonstrated instrumentally.

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