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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2012 1
2014 3
2015 1
2016 2
2017 5
2018 4
2019 2
2020 2
2021 3
2022 1
2023 1
2024 1

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20 results

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Page 1
Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective.
Dawkins HJS, Draghia-Akli R, Lasko P, Lau LPL, Jonker AH, Cutillo CM, Rath A, Boycott KM, Baynam G, Lochmüller H, Kaufmann P, Le Cam Y, Hivert V, Austin CP; International Rare Diseases Research Consortium (IRDiRC). Dawkins HJS, et al. Among authors: le cam y. Clin Transl Sci. 2018 Jan;11(1):11-20. doi: 10.1111/cts.12501. Epub 2017 Oct 23. Clin Transl Sci. 2018. PMID: 28796411 Free PMC article. Review. No abstract available.
Towards Achieving Equity and Innovation in Newborn Screening across Europe.
Sikonja J, Groselj U, Scarpa M, la Marca G, Cheillan D, Kölker S, Zetterström RH, Kožich V, Le Cam Y, Gumus G, Bottarelli V, van der Burg M, Dekkers E, Battelino T, Prevot J, Schielen PCJI, Bonham JR. Sikonja J, et al. Among authors: le cam y. Int J Neonatal Screen. 2022 May 6;8(2):31. doi: 10.3390/ijns8020031. Int J Neonatal Screen. 2022. PMID: 35645285 Free PMC article. Review.
Medicines Adaptive Pathways to Patients: Why, When, and How to Engage?
Eichler HG, Bedlington N, Boudes M, Bouvy JC, Broekmans AW, Cerreta F, Faulkner SD, Forda SR, Joos A, Le Cam Y, Mayer MH, Pirard V, Corriol-Rohou S; ADAPT SMART Consortium. Eichler HG, et al. Among authors: le cam y. Clin Pharmacol Ther. 2019 May;105(5):1148-1155. doi: 10.1002/cpt.1121. Epub 2018 Sep 11. Clin Pharmacol Ther. 2019. PMID: 29901216 Free PMC article. Review.
Reply to E. Vicente et al.
Rath A, Lambert DM, Olry A, Rodwell C, Le Cam Y. Rath A, et al. Among authors: le cam y. Eur J Hum Genet. 2021 Jun;29(6):1034-1035. doi: 10.1038/s41431-020-00764-y. Epub 2020 Dec 1. Eur J Hum Genet. 2021. PMID: 33262444 Free PMC article. No abstract available.
Our greatest untapped resource: our patients.
Bolz-Johnson M, Kenny T, Le Cam Y, Hernando I. Bolz-Johnson M, et al. Among authors: le cam y. J Community Genet. 2021 Apr;12(2):241-246. doi: 10.1007/s12687-021-00524-5. Epub 2021 Apr 21. J Community Genet. 2021. PMID: 33884523 Free PMC article.
Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health Framework.
Baynam G, Bowman F, Lister K, Walker CE, Pachter N, Goldblatt J, Boycott KM, Gahl WA, Kosaki K, Adachi T, Ishii K, Mahede T, McKenzie F, Townshend S, Slee J, Kiraly-Borri C, Vasudevan A, Hawkins A, Broley S, Schofield L, Verhoef H, Groza T, Zankl A, Robinson PN, Haendel M, Brudno M, Mattick JS, Dinger ME, Roscioli T, Cowley MJ, Olry A, Hanauer M, Alkuraya FS, Taruscio D, Posada de la Paz M, Lochmüller H, Bushby K, Thompson R, Hedley V, Lasko P, Mina K, Beilby J, Tifft C, Davis M, Laing NG, Julkowska D, Le Cam Y, Terry SF, Kaufmann P, Eerola I, Norstedt I, Rath A, Suematsu M, Groft SC, Austin CP, Draghia-Akli R, Weeramanthri TS, Molster C, Dawkins HJS. Baynam G, et al. Among authors: le cam y. Adv Exp Med Biol. 2017;1031:55-94. doi: 10.1007/978-3-319-67144-4_4. Adv Exp Med Biol. 2017. PMID: 29214566 Review.
The evolution of adaptiveness: balancing speed and evidence.
Eichler HG, Barker R, Bedlington N, Bouvy JC, Broekmans AW, Bucsics A, Cerreta F, Corriol-Rohou S, Granados A, Le Cam Y, Schuurman A. Eichler HG, et al. Among authors: le cam y. Nat Rev Drug Discov. 2018 Dec;17(12):845-846. doi: 10.1038/nrd.2018.90. Epub 2018 Jul 6. Nat Rev Drug Discov. 2018. PMID: 29977052 No abstract available.
Strengthening health systems for access to gene therapy in rare genetic disorders.
Bhatia S, Le Cam Y, Carrion J, Diamond L, Fennessy P, Gassman S, Gutzwiller F, Kagan S, Pankevich D, Young Maloney J, Mahadev N, Schulz M, Wong-Rieger D, Morgese P. Bhatia S, et al. Among authors: le cam y. Mol Ther Methods Clin Dev. 2024 Mar 13;32(2):101220. doi: 10.1016/j.omtm.2024.101220. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38516694 Free PMC article. No abstract available.
Patients, payers and developers of Orphan Medicinal Products: lessons learned from 10 years' multi-stakeholder dialogue on improving access in Europe via MoCA.
Cavaller-Bellaubi M, Hughes-Wilson W, Kubinová Š, Van de Casteele M, Van Lente EJ, Degortes E, Pontén J, Eichler HG, Le Cam Y, Boselli S, Bucsics A. Cavaller-Bellaubi M, et al. Among authors: le cam y. Orphanet J Rare Dis. 2023 Jun 12;18(1):144. doi: 10.1186/s13023-023-02774-7. Orphanet J Rare Dis. 2023. PMID: 37308991 Free PMC article.
20 results