Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

40 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Développement des médicaments en pédiatrie : défis existants et recommandations.
Kaguelidou F, Ouèdraogo M, Treluyer JM, Le Jeunne C, Annereau M, Blanc P, Bureau S, Ducassou S, Fiquet B, Flamein F, Gaillard S, Hankard R, Laugel V, Laurent C, Levy C, Marquet T, Polak M, Portefaix A, Vassal G. Kaguelidou F, et al. Among authors: portefaix a. Therapie. 2023 Jan-Feb;78(1):95-104. doi: 10.1016/j.therap.2022.12.002. Epub 2022 Dec 5. Therapie. 2023. PMID: 36543724 French. No abstract available.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.
Mercuri E, Deconinck N, Mazzone ES, Nascimento A, Oskoui M, Saito K, Vuillerot C, Baranello G, Boespflug-Tanguy O, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Gerber M, Gorni K, Khwaja O, Kletzl H, Scalco RS, Staunton H, Yeung WY, Martin C, Fontoura P, Day JW; SUNFISH Study Group. Mercuri E, et al. Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7. Lancet Neurol. 2022. PMID: 34942136
High flow nasal cannula (HFNC) versus nasal continuous positive airway pressure (nCPAP) for the initial respiratory management of acute viral bronchiolitis in young infants: a multicenter randomized controlled trial (TRAMONTANE study).
Milési C, Essouri S, Pouyau R, Liet JM, Afanetti M, Portefaix A, Baleine J, Durand S, Combes C, Douillard A, Cambonie G; Groupe Francophone de Réanimation et d’Urgences Pédiatriques (GFRUP). Milési C, et al. Among authors: portefaix a. Intensive Care Med. 2017 Feb;43(2):209-216. doi: 10.1007/s00134-016-4617-8. Epub 2017 Jan 26. Intensive Care Med. 2017. PMID: 28124736 Clinical Trial.
A multicenter randomized controlled trial of a 3-L/kg/min versus 2-L/kg/min high-flow nasal cannula flow rate in young infants with severe viral bronchiolitis (TRAMONTANE 2).
Milési C, Pierre AF, Deho A, Pouyau R, Liet JM, Guillot C, Guilbert AS, Rambaud J, Millet A, Afanetti M, Guichoux J, Genuini M, Mansir T, Bergounioux J, Michel F, Marcoux MO, Baleine J, Durand S, Durand P, Dauger S, Javouhey E, Leteurtre S, Brissaud O, Renolleau S, Portefaix A, Douillard A, Cambonie G; GFRUP Respiratory Study Group. Milési C, et al. Among authors: portefaix a. Intensive Care Med. 2018 Nov;44(11):1870-1878. doi: 10.1007/s00134-018-5343-1. Epub 2018 Oct 21. Intensive Care Med. 2018. PMID: 30343318 Clinical Trial.
Type I Interferon in Children with Viral or Bacterial Infections.
Trouillet-Assant S, Viel S, Ouziel A, Boisselier L, Rebaud P, Basmaci R, Droz N, Belot A, Pons S, Brengel-Pesce K, Gillet Y, Javouhey E; Antoine Study Group. Trouillet-Assant S, et al. Clin Chem. 2020 Jun 1;66(6):802-808. doi: 10.1093/clinchem/hvaa089. Clin Chem. 2020. PMID: 32359149
Agreement between a regional pharmacovigilance centre and an adjudication committee regarding adverse drug reactions on a cohort of hospitalised children.
Pages N, Bounabi A, Boussaha I, Ndiaye M, Portefaix A, Simeon G, Guy C, Stagnara J, Paret N, Vial T, Eftekhari P, Floret D, Gajdos V, Langhendries JP, Bleyzac N, Alberti C, Jacqz-Aigrain E, Nguyen KA, Kassai B; EREMI Group. Pages N, et al. Among authors: portefaix a. Therapie. 2022 Jul-Aug;77(4):397-404. doi: 10.1016/j.therap.2021.12.014. Epub 2021 Dec 24. Therapie. 2022. PMID: 34998623
Paediatric drug development and evaluation: Existing challenges and recommendations.
Kaguelidou F, Ouèdraogo M, Treluyer JM, Le Jeunne C, Annereau M, Blanc P, Bureau S, Ducassou S, Fiquet B, Flamein F, Gaillard S, Hankard R, Laugel V, Laurent C, Levy C, Marquet T, Polak M, Portefaix A, Vassal G. Kaguelidou F, et al. Among authors: portefaix a. Therapie. 2023 Jan-Feb;78(1):105-114. doi: 10.1016/j.therap.2022.11.010. Epub 2022 Dec 5. Therapie. 2023. PMID: 36528416
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial.
Frishberg Y, Deschênes G, Groothoff JW, Hulton SA, Magen D, Harambat J, Van't Hoff WG, Lorch U, Milliner DS, Lieske JC, Haslett P, Garg PP, Vaishnaw AK, Talamudupula S, Lu J, Habtemariam BA, Erbe DV, McGregor TL, Cochat P; study collaborators. Frishberg Y, et al. Clin J Am Soc Nephrol. 2021 Jul;16(7):1025-1036. doi: 10.2215/CJN.14730920. Epub 2021 May 13. Clin J Am Soc Nephrol. 2021. PMID: 33985991 Free PMC article. Clinical Trial.
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschênes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Garrelfs SF, et al. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712. N Engl J Med. 2021. PMID: 33789010 Clinical Trial.
40 results