20192020

Research output per year

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Research Output

Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases

Sheean, M. E., Malikova, E., Duarte, D., Capovilla, G., Fregonese, L., Hofer, M. P., Magrelli, A., Mariz, S., Mendez-Hermida, F., Nistico, R., Leest, T., Sipsas, N. V., Tsigkos, S., Vitezic, D., Larsson, K., Sepodes, B. & Stoyanova-Beninska, V., Feb 2020, In : Drug Discovery Today. 25, 2, p. 274-291 18 p.

Research output: Contribution to journalReview article

  • Defining orphan conditions in the context of the European orphan regulation: challenges and evolution

    O'Connor, D. J., Sheean, M. E., Hofer, M. P., Tsigkos, S., Mariz, S., Fregonese, L., Larsson, K., Hivert, V., Westermark, K., Naumann-Winter, F., Stoyanova-Beninska, V., Barišić, I., Capovilla, G., Magrelli, A. & Sepodes, B., Jul 2019, In : Nature Reviews Drug Discovery. 18, 7, p. 479-480 2 p.

    Research output: Contribution to journalArticle

    European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials

    Haberkamp, M., Moseley, J., Athanasiou, D., de Andres-Trelles, F., Elferink, A., Rosa, M. M. & Magrelli, A., Jul 2019, In : Neuromuscular Disorders. 29, 7, p. 514-516 3 p.

    Research output: Contribution to journalArticle

  • Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy

    López-Bastida, J., Ramos-Goñi, J. M., Aranda-Reneo, I., Taruscio, D., Magrelli, A. & Kanavos, P., Jun 26 2019, In : Orphanet Journal of Rare Diseases. 14, 1, p. 154

    Research output: Contribution to journalArticle