Authors' objectives: To assess the available evidence on the efficacy, safety and coverage related aspects regarding the use of miglustat in patients with Niemann-Pick disease type C.

Authors' recommendations: There is evidence of moderate methodological quality based on a randomized study, one systematic review and case series with few patients showing that miglustat stabilizes or discretely improves neurological progression measured as speed of saccadic movements and swallowing in Niemann-Pick disease type C patients with neurologic manifestations. This was observed both in children and in children above 12 years old. Given the chronic and progressive neurodegenerative nature of the disease and its low frequency, it is difficult to find evidence of high quality or with important targets. In this type of diseases, stabilization or a decrease in disease progression are likely to be the expected objectives for the long-term specific treatment of the disease.

Project Status: Completed

Year Published: 2013

URL for published report: https://www.iecs.org.ar/home-ets/?cod_publicacion=1562&familia=5&origen_publicacion=buscador

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Argentina

Organisation Name: Institute for Clinical Effectiveness and Health Policy

Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA

Contact Name: info@iecs.org.ar

Contact Email: info@iecs.org.ar

Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)