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

ABSTRACT:

Importance

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.

Objective

To determine if miglustat therapy is associated with stabilized swallowing dysfunction in individuals with NPC1.

Design, setting, and participants

Patients with confirmed NPC1 diagnoses were evaluated in a single-center cohort study of NPC1 from April 1997 to November 2019. Longitudinal data from

SUBMITTER: Solomon BI 

PROVIDER: S-EPMC7489403 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature