Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases

The following new articles have just been published in Orphanet Journal of Rare Diseases REVIEW Patient and observer reported outcome measures to evaluate health-related quality of life in inherited metabolic diseases: a scoping review Carlota Pascoal, Sandra Brasil, Rita Francisco, Dorinda Marques-da-Silva, Agnes Rafalko, Jaak Jaeken, Paula A. Videira, Luísa Barros and Vanessa dos Reis Ferreira Orphanet Journal of Rare Diseases 2018, 13:215 | Published on: 28 November 2018 Full Text | PDF RESEARCH Oncologic orphan drugs approved in the EU – do clinical trial data correspond with real-world effectiveness? Yvonne Schuller, Marieke Biegstraaten, Carla E. M. Hollak, Heinz-Josef Klümpen, Christine C. Gispen-de Wied and Violeta Stoyanova-Beninska Orphanet Journal of Rare Diseases 2018, 13:214 | Published on: 28 November 2018 Full Text | PDF

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