Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher
disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are
required to establish the long term efficacy, safety and tolerability of miglustat in
maintaining diseases stability after a switch from ERT.
This is a phase II randomized controlled study of miglustat in adult and juvenile patients
with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to
either treatment with miglustat or to a non-treatment group. Both groups will follow an
identical visit schedule.
Miglustat / OGT 918 in the Treatment of Cystic Fibrosis
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane
conductance regulator (CFTR). The purpose of the study is to investigate the effects of
miglustat on CFTR function in cystic fibrosis patients.
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute
infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff
disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that
is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits
the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical
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