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Current Medical Research and Opinion Volume 37, 2021 - Issue 6

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Orphan Drugs

Value-based decision-making for orphan drugs with multiple criteria decision analysis: burosumab for the treatment of X-linked hypophosphatemia

Björn Vandewallea Exigo Consultores, Lisbon, PortugalCorrespondence[email protected]

Miguel Amorima Exigo Consultores, Lisbon, Portugal

Diogo Ramosa Exigo Consultores, Lisbon, Portugal

Sofia Azevedoa Exigo Consultores, Lisbon, Portugal

Inês Alvesb Associação Nacional de Displasias Ósseas, Évora, Portugal

Telma Franciscoc Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal

Helena Pintod Centro Hospitalar Universitário de São João, Porto, Portugal

Sérgio Sousae Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal

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Pages 1021-1030 | Received 26 Nov 2020, Accepted 14 Mar 2021, Published online: 01 Apr 2021

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https://doi.org/10.1080/03007995.2021.1904861
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M. I. Luis Yanes, M. Diaz-Curiel, P. Peris, C. Vicente, S. Marin, M. Ramon-Krauel, J. Hernandez, J. J. Broseta, L. Espinosa, S. Mendizabal, L. Perez-Sukia, V. Martínez, C. Palazón, J. A. Piñero, M. A. Calleja, J. Espin, R. Arborio-Pinel & G. Ariceta. (2022) Health-related quality of life of X-linked hypophosphatemia in Spain. Orphanet Journal of Rare Diseases 17:1.
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Nadja Fratzl‐Zelman, Markus A. Hartmann, Sonja Gamsjaeger, Stamatia Rokidi, Eleftherios P. Paschalis, Stéphane Blouin & Jochen Zwerina. (2022) Bone Matrix Mineralization and Response to Burosumab in Adult Patients With X‐Linked Hypophosphatemia: Results From the Phase 3, Single‐Arm International Trial. Journal of Bone and Mineral Research 37:9, pages 1665-1678.
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