Qualitative Study of the Patient Experience with Venglustat for Gaucher Disease Type 3 in a Phase 2 Open-Label, Multicenter, Multinational Study (LEAP)

Author:

Schiffmann RaphaelORCID,Mengel EugenORCID,Wallace Mary,Rochmann Camille,Turnbull James,Krupnick Robert,Gwaltney Chad,Pulikottil-Jacob Ruth,Batsu Isabela,Zheng Riliang,Hamed Alaa

Funder

Sanofi

Publisher

Springer Science and Business Media LLC

Reference20 articles.

1. Grabowski GA. Gaucher disease and other storage disorders. Hematology Am Soc Hematol Educ Program. 2012;2012:13–8.

2. Schiffmann R, Sevigny J, Rolfs A, et al. The definition of neuronopathic Gaucher disease. J Inherit Metab Dis. 2020;43(5):1056–9.

3. Grabowski GA, Zimran A, Ida H. Gaucher disease types 1 and 3: phenotypic characterization of large populations from the ICGG Gaucher Registry. Am J Hematol. 2015;90(Suppl 1):S12–8.

4. Ramaswami U, Mengel E, Berrah A, et al. Throwing a spotlight on under-recognized manifestations of Gaucher disease: pulmonary involvement, lymphadenopathy and gaucheroma. Mol Genet Metab. 2021;133(4):335–44.

5. Grabowski G PG, Kolodny EH. Gaucher disease. 2013. In: OMMID: The Online Metabolic and Molecular Bases of Inherited Disease [Internet]. New York, NY: McGraw-Hill. Available from: https://ommbid.mhmedical.com/ommbid-index.aspx. Accessed 15 Apr 2024.

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