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Orphanet Journal of Rare Diseases

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de las Heras J, Cebolla J, de Pedro S, Gomez-Barrera M, Vitoria I

 

 

Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: A systematic review across all paediatric age groups
Orphanet J Rare Dis. 2026. doi: 10.1186/s13023-026-04221-9

de Andrés-Nogales F, Cruz E, Calleja MA, Delgado O, Gorgas Torner MQ, Espín J, Mestre-Ferrándiz J, Palau F, Ancochea A, Arce R, Domínguez-Hernández R, Casado MA and on behalf of the FinMHU-MCDA Group

 

A Multi-Stakeholder Multicriteria Decision Analysis for the Reimbursement of Orphan Drugs (FinMHU-MCDA study)
Orphanet J Rare Dis. 2021 Apr 26;16(1):186