Associated Papers
Plasma glucosylsphingosine correlations with baseline disease burden and response to eliglustat in two clinical trials of previously untreated adults with Gaucher disease type 1.
Peterschmitt MJ et al.
Molecular Genetics and Metabolism, 2023
Incremental biomarker and clinical outcomes after switch from enzyme therapy to eliglustat substrate reduction therapy in Gaucher disease.
Kleytman N et al.
Molecular Genetics and Metabolism Reports, 2021
Real-world effectiveness of eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
Mistry PK et al.
American Journal of Hematology, 2020
Genetic Risk Factors for Essential Tremor: A Review.
Siokas V et al.
Tremor and Other Hyperkinetic Movements (new York, N.y.), 2020
32775018 (PubMed )
Supplement: tohm-10-1-67-s2.pdf
Organoid and pluripotent stem cells in Parkinson's disease modeling: an expert view on their value to drug discovery.
Marotta N et al.
Expert Opinion on Drug Discovery, 2020
Glucosylsphingosine but not Saposin C, is the target antigen in Gaucher disease-associated gammopathy.
Nair S et al.
Molecular Genetics and Metabolism, 2020
Reduction of large soft-tissue Gaucheromas with substrate reduction therapy.
Mahajan N et al.
Journal of Inherited Metabolic Disease, 2020
A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months.
Ida H et al.
Blood Cells, Molecules & Diseases, 2016
Gaucher disease and its treatment options.
Bennett LL et al.
The Annals of Pharmacotherapy, 2013
A Japanese Patient with Gaucher Disease Treated with the Oral Drug Eliglustat as Substrate Reducing Therapy.
Komada N et al.
Case Reports in Gastroenterology, 0
Select a paper to view details