E-LIB Suche - Ergebnisse für: Le Cam, Yann

1

Strengthening health systems for access to gene therapy in ..:

Bhatia, Sonal ; Le Cam, Yann ; Carrion, Juan...
Molecular Therapy - Methods & Clinical Development. 32 (2024) 2 - p. 101220 , 2024

Link: https://doi.org/10.1016/..

2

Time to diagnosis and determinants of diagnostic delays of ..:

Faye, Fatoumata ; Crocione, Claudia ; Anido de Peña, Roberta...
European Journal of Human Genetics. , 2024

Link: https://doi.org/10.1038/..

3

Patients, payers and developers of Orphan Medicinal Product..:

Cavaller-Bellaubi, Maria ; Hughes-Wilson, Wills ; Kubinová, Šárka...
Orphanet Journal of Rare Diseases. 18 (2023) 1 - p. , 2023

Link: https://doi.org/10.1186/..

4

Towards Achieving Equity and Innovation in Newborn Screenin..:

Sikonja, Jaka ; Groselj, Urh ; Scarpa, Maurizio...
International Journal of Neonatal Screening. 8 (2022) 2 - p. 31 , 2022

Link: https://doi.org/10.3390/..

5

Our greatest untapped resource: our patients:

Bolz-Johnson, Matt ; Kenny, Tom ; Le Cam, Yann.
Journal of Community Genetics. 12 (2021) 2 - p. 241-246 , 2021

Link: https://doi.org/10.1007/..

6

Orphan Medicine Incentives: How to Address the Unmet Needs ..:

Aartsma-Rus, Annemieke ; Dooms, Marc ; Le Cam, Yann
Frontiers in Pharmacology. 12 (2021) - p. , 2021

Link: https://doi.org/10.3389/..

7

Priorities on rare cancers' policy in National Cancer Contr..:

Prades, Joan ; Weinman, Ariane ; Le Cam, Yann...
Journal of Cancer Policy. 24 (2020) - p. 100222 , 2020

Link: https://doi.org/10.1016/..

8

Reply to E. Vicente et al:

Rath, Ana ; Lambert, Deborah M. ; Olry, Annie..
European Journal of Human Genetics. 29 (2020) 6 - p. 1034-1035 , 2020

Link: https://doi.org/10.1038/..

9

Expert by Experience: Valuing Patient Engagement in Healthc..:

, In: Patient Engagement; Organizational Behaviour in Healthcare,

Le Cam, Yann ; Bolz-Johnson, Matthew - p. 233-267 , 2019

Link: https://doi.org/10.1007/..

10

Estimating cumulative point prevalence of rare diseases: an..:

Nguengang Wakap, Stéphanie ; Lambert, Deborah M. ; Olry, Annie...
European Journal of Human Genetics. 28 (2019) 2 - p. 165-173 , 2019

Link: https://doi.org/10.1038/..

11

Medicines Adaptive Pathways to Patients: Why, When, and How..:

Eichler, Hans‐Georg ; Bedlington, Nicola ; Boudes, Mathieu...
Clinical Pharmacology & Therapeutics. 105 (2018) 5 - p. 1148-1155 , 2018

Link: https://doi.org/10.1002/..

12

The International Rare Diseases Research Consortium: Polici..:

on behalf of the IRDiRC Consortium Assembly ; Lochmüller, Hanns ; Torrent i Farnell, Josep...
European Journal of Human Genetics. 25 (2017) 12 - p. 1293-1302 , 2017

Link: https://doi.org/10.1038/..

13

Progress in Rare Diseases Research 2010–2016: An IRDiRC Per..:

Dawkins, Hugh J.S. ; Draghia‐Akli, Ruxandra ; Lasko, Paul...
Clinical and Translational Science. 11 (2017) 1 - p. 11-20 , 2017

Link: https://doi.org/10.1111/..

14

Recommendations from the European Working Group for Value A..:

Annemans, Lieven ; Aymé, Ségolène ; Le Cam, Yann...
Orphanet Journal of Rare Diseases. 12 (2017) 1 - p. , 2017

Link: https://doi.org/10.1186/..

15

'IRDiRC Recognized Resources': a new mechanism to support s..:

on behalf of the IRDiRC Scientific Committees ; Lochmüller, Hanns ; Le Cam, Yann...
European Journal of Human Genetics. 25 (2016) 2 - p. 162-165 , 2016

Link: https://doi.org/10.1038/..