New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

Overview of attention for article published in Therapeutics and Clinical Risk Management, November 2009

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Overview of clinical, molecular, and therapeutic features of Niemann–Pick disease (types A, B, and C): Focus on therapeutic approaches

Article in Cell Biochemistry and Function (May 2024)

Long-Term Evaluation of Biomarkers in the Czech Cohort of Gaucher Patients

Article in International Journal of Molecular Sciences (September 2023)

Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review

Article in European Journal of Neurology (June 2023)