Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis.

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Published in Mol Genet Metab on May 03, 2009

Authors

Gustavo H B Maegawa1, Paul L M van Giersbergen, Sandra Yang, Brenda Banwell, Christopher P Morgan, Jasper Dingemanse, Cynthia J Tifft, Joe T R Clarke

Author Affiliations

1: Division of Clinical and Metabolic Genetics, Hospital for Sick Children, Toronto, Ont., Canada.

Associated clinical trials:

Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G) | NCT02030015

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