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Author Details

Yann Le Cam
2012
23
12
PMIDPaper TitleJournal TitlePublished Year
37308991Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA.Orphanet J Rare Dis2023
35645285Towards Achieving Equity and Innovation in Newborn Screening across Europe.Int J Neonatal Screen2022
33262444Reply to E. Vicente et al.Eur J Hum Genet2021
33884523Our greatest untapped resource: our patients.J Community Genet2021
34975469Orphan 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).Front Pharmacol2021
31527858Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database.Eur J Hum Genet2020
29901216Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?Clin Pharmacol Ther2019
28796411Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective.Clin Transl Sci2018
29911566[French model for Europe and Europe as a model for France in the field of rare diseases].Med Sci (Paris)2018
29977052The evolution of adaptiveness: balancing speed and evidence.Nat Rev Drug Discov2018
27782107'IRDiRC Recognized Resources': a new mechanism to support scientists to conduct efficient, high-quality research for rare diseases.Eur J Hum Genet2017
28283046Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL).Orphanet J Rare Dis2017
29158551The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.Eur J Hum Genet2017
29214566Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health Framework.Adv Exp Med Biol2017
26929314A model for active and healthy ageing with a rare genetic disease: cystic fibrosis.Eur Respir J2016
26911987The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference Networks.Orphanet J Rare Dis2016
25537360The EuroBioBank Network: 10 years of hands-on experience of collaborative, transnational biobanking for rare diseases.Eur J Hum Genet2015
25669457From adaptive licensing to adaptive pathways: delivering a flexible life-span approach to bring new drugs to patients.Clin Pharmacol Ther2015
24513034Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives.Orphanet J Rare Dis2014
24922305European Reference Networks for Rare Diseases: the vision of patients.Blood Transfus2014
24503588EUROPLAN: a project to support the development of national plans on rare diseases in Europe.Public Health Genomics2013
22258596Leap year: Rare day to highlight rare diseases.Nature2012
23293582Involvement of patient organisations in research and development of orphan drugs for rare diseases in europe.Mol Syndromol2012
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Collaborators

Hanns Lochm??ller
Children's Hospital of Eastern Ontario Research Institute
Co-authored papers 5
Petra Kaufmann
Novartis Gene Therapies
Co-authored papers 4
Hugh Dawkins
The University of Notre Dame Australia
Co-authored papers 4
Paul Lasko
McGill University. Montreal
Co-authored papers 4
Christopher P Austin
St. George's University Medical School
Co-authored papers 4
Ana Rath
INSERM
Co-authored papers 4
Kym M Boycott
Children's Hospital of Eastern Ontario
Co-authored papers 4
Gareth Baynam
King Edward Memorial Hospital
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Kate Bushby
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Annie Olry
INSERM
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Manuel Posada de la Paz
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Victoria Hedley
John Walton Muscular Dystrophy Research Centre, Newcastle University
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Ruxandra Draghia-Akli
The Janssen Pharmaceutical Companies of Johnson & Johnson
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Domenica Taruscio
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Lyn Schofield
King Edward Memorial Hospital for Women Perth
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Kenneth A Oye
Massachusetts Institute of Technology
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S??gol??ne Aym??
Paris Brain Institute-ICM, Inserm U 7, CNRS UMR 7225, Sorbonne Universite
Co-authored papers 1
Takeya Adachi
Keio University School of Medicine
Co-authored papers 1
Tarun Weeramanthri
University of Western Australia
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Daria Julkowska
Institut National de la Sante et de la Recherche Medicale
Co-authored papers 1
John S Mattick
Australia UNSW RNA Institute
Co-authored papers 1
Irene Norstedt
Co-authored papers 1
Rachel Thompson
Children's Hospital of Eastern Ontario Research Institute
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Jack Goldblatt
King Edward Memorial Hospital for Women
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Ken Ishii
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Melissa A Haendel
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Alessandra Renieri
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Mark J Cowley
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Release 1.2