Results 141 to 150 of about 2,627 (188)

Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial

Molecular Genetics and Metabolism, 2010
A randomized, controlled trial of miglustat indicated that miglustat (Zavesca®) stabilized neurological disease over 12 months in adult and juvenile patients with Niemann-Pick disease type C (NP-C).
Marc C. Patterson, Larry Abel, Ruben Giorgino   +2 more
exaly   +2 more sources

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Miglustat

Drugs, 2012
Paul L McCormack
exaly   +3 more sources

A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference [PDF]

Journal of Cystic Fibrosis, 2012
BACKGROUND: Preclinical data suggest that miglustat could restore the function of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis cells. METHODS: Single-center, randomized, double-blind, placebo-controlled, crossover Phase
Jasper Dingemanse, Teresinha Leal, Anissa Leonard   +2 more
exaly   +2 more sources

Normalisation of brain spectroscopy findings in Niemann–Pick disease type C patients treated with miglustat [PDF]

Journal of Neurology, 2016
International audienceNiemann-Pick disease type C (NP-C) is a fatal progressive neurolipidosis involving neuronal storage of cholesterol and gangliosides.
Elsa Kaphan, Chabrol Brigitte, Christine Tranchant   +2 more
exaly   +3 more sources

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study

Molecular Genetics and Metabolism, 2009
Miglustat has been shown to stabilize disease progression in children, juveniles and adults with Niemann-Pick disease type C (NP-C), a rare genetic disorder characterized by progressive neurological deterioration.
Mark Walterfang, Mercè Pineda i Marfà, E Mengel   +2 more
exaly   +2 more sources