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Patryk Lipiński

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Review Open access Sep 2026

Miglustat in Neuronopathic Lysosomal Storage Disorders: Biological Rationale, Clinical Evidence, and Limits of Repurposing

Neuronopathic lysosomal storage disorders remain difficult to treat because conventional enzyme replacement therapies generally do not reach therapeutically meaningful concentrations in the central nervous system. Miglustat (1,5-(butylimino)-1,5-dideoxy-D-glucitol; N-butyl-deoxynojirimycin) is an orally administered im...

Patryk Lipiński, Grzegorz Węgrzyn · 0 citations
Review Open access Sep 2026

Ambroxol in Gaucher disease and GBA1-related Parkinson disease: an updated systematic review.

BACKGROUND Ambroxol (ABX) is a central nervous system (CNS)-penetrant compound that can increase glucocerebrosidase activity and has been investigated in Gaucher disease (GD) and GBA1-related Parkinson disease (GBA1-PD). METHODS This PRISMA 2020 systematic review and narrative synthesis searched PubMed/MEDLINE, Europ...

Patryk Lipiński, A. Jezela-Stanek, A. Tylki-Szymańska · 0 citations

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