Last Updated: 09/26/2026
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Found 49 publications
Emerging biomarkers in Gaucher disease.
Journal: Advances in clinical chemistry
Published: January 16, 2025
Challenges in Gaucher disease: Perspectives from an expert panel.
Journal: Molecular genetics and metabolism
Published: September 28, 2024
The international cooperative Gaucher group (ICCG) Gaucher registry.
Journal: Best practice & research. Clinical haematology
Published: October 04, 2023
Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent.
Journal: JAMA network open
Published: June 21, 2023
The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158.
Journal: Journal of clinical medicine
Published: January 20, 2023
Cancer risk and gammopathies in 2123 adults with Gaucher disease type 1 in the International Gaucher Group Gaucher Registry.
Journal: American journal of hematology
Published: May 19, 2022
Physiologically-Based Pharmacokinetic Model Development, Validation, and Application for Prediction of Eliglustat Drug-Drug Interactions.
Journal: Clinical pharmacology and therapeutics
Published: May 16, 2022
Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: an interim analysis of real-world data from a multinational drug registry (TALIAS).
Journal: Orphanet journal of rare diseases
Published: December 20, 2021
The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?
Journal: Molecular genetics and metabolism
Published: November 30, 2021
Screening, patient identification, evaluation, and treatment in patients with Gaucher disease: Results from a Delphi consensus.
Journal: Molecular genetics and metabolism
Published: July 27, 2021
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials.
Journal: Orphanet journal of rare diseases
Published: July 14, 2021
Gaucher disease - more than just a rare lipid storage disease.
Journal: Journal of molecular medicine (Berlin, Germany)
Published: July 01, 2021
Last Updated: 09/26/2026