Associated Papers
Genotype Meets Phenotype: Unraveling Gaucher's Genetic Landscape in the Indian Population.
Powline DG et al.
Cureus, 2025
Rapid and long-lasting efficacy of high-dose ambroxol therapy for neuronopathic Gaucher disease: A case report and literature review.
Higashi K et al.
Molecular Genetics & Genomic Medicine, 2024
38553911 (PubMed )
Supplement: MGG3-12-e2427-s001.docx
Exploring the efficacy and safety of Ambroxol in Gaucher disease: an overview of clinical studies.
Mohamed FE et al.
Frontiers in Pharmacology, 2024
Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial.
Peterschmitt MJ et al.
Journal of Parkinson's Disease, 2022
Current and emerging pharmacotherapy for Gaucher disease in pediatric populations.
Sam R et al.
Expert Opinion on Pharmacotherapy, 2021
Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy.
Kim YM et al.
Journal of Medical Genetics, 2020
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