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Found 10 publications

Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data.

Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment.

Quality of life of 22 patients with type 1 Gaucher disease after enzyme replacement therapy.

Liver transplantation for Gaucher disease presenting as neonatal cholestasis: Case report and literature review.

Impact of hepatic and renal impairment on the pharmacokinetics and tolerability of eliglustat therapy for Gaucher disease type 1.

Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy.

Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial.

Splenectomy in Gaucher Disease: A Call for Minimally Invasive Surgery.

Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.

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