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Frontiers in Pharmacology|January 3, 2022
Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group)Annemieke Aartsma-Rus, Marc Dooms, Yann Le Cam
Journal of Community Genetics|April 22, 2021
Our greatest untapped resource: our patientsMatt Bolz-Johnson, Tom Kenny, Yann Le Cam, et al.
Orphanet Journal of Rare Diseases|February 26, 2016
The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference NetworksTeresinha Evangelista, Victoria Hedley, Antonio Atalaia, et al.
Orphanet Journal of Rare Diseases|February 12, 2014
Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectivesDavid Uguen, Thomas Lönngren, Yann Le Cam, et al.
European Journal of Human Genetics : EJHG|September 19, 2019
Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet databaseStéphanie Nguengang Wakap, Deborah M Lambert, Annie Olry, et al.
European Journal of Human Genetics : EJHG|October 27, 2016
'IRDiRC Recognized Resources': a new mechanism to support scientists to conduct efficient, high-quality research for rare diseasesHanns Lochmüller, Yann Le Cam, Anneliene H Jonker, et al.
Orphanet Journal of Rare Diseases|June 12, 2023
Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCAMaria Cavaller-Bellaubi, Wills Hughes-Wilson, Šárka Kubinová, et al.
European Journal of Human Genetics : EJHG|November 22, 2017
The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impactHanns Lochmüller, Josep Torrent I Farnell, Yann Le Cam, et al.
European Journal of Human Genetics : EJHG|May 16, 2024
Time to diagnosis and determinants of diagnostic delays of people living with a rare disease: results of a Rare Barometer retrospective patient surveyFatoumata Faye, Claudia Crocione, Roberta Anido de Peña, et al.
Orphanet Journal of Rare Diseases|March 12, 2017
Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)Lieven Annemans, Ségolène Aymé, Yann Le Cam, et al.
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