Özlem Göker-Alpan

Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey

P. Kishnani, P. Deegan, A. Zimran, 2017, Molecular genetics and metabolism reports.

Screening, patient identification, evaluation, and treatment in patients with Gaucher disease: Results from a Delphi consensus.

M. Balwani, P. Kishnani, M. Wasserstein, 2021, Molecular genetics and metabolism.

Exploring the patient journey to diagnosis of Gaucher disease from the perspective of 212 patients with Gaucher disease and 16 Gaucher expert physicians.

J. Szer, B. Bembi, A. Mehta, 2017, Molecular genetics and metabolism.

Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)

S. Revel-Vilk, D. Hughes, J. Botha, 2022, Journal of clinical medicine.

Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

L. Longworth, N. Belmatoug, D. Hughes, 2022, Orphanet Journal of Rare Diseases.