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2015 1
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2023 10
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Page 1
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
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.
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Chiriboga CA, Bruno C, Duong T, Fischer D, Mercuri E, Kirschner J, Kostera-Pruszczyk A, Jaber B, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scalco RS, Wagner KR, Muntoni F; JEWELFISH Study Group. Chiriboga CA, et al. Neurol Ther. 2023 Apr;12(2):543-557. doi: 10.1007/s40120-023-00444-1. Epub 2023 Feb 13. Neurol Ther. 2023. PMID: 36780114 Free PMC article.
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.
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.
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.
Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children.
Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Sas DJ, et al. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8. Genet Med. 2022. PMID: 34906487 Free article. Clinical Trial.
Diagnostic accuracy of a calibrated abdominal compression to predict fluid responsiveness in children.
Jacquet-Lagrèze M, Tiberghien N, Evain JN, Hanna N, Courtil-Teyssedre S, Lilot M, Baudin F, Chardonnal L, Bompard D, Koffel C, Portefaix A, Javouhey E, Fellahi JL. Jacquet-Lagrèze M, et al. Among authors: portefaix a. Br J Anaesth. 2018 Dec;121(6):1323-1331. doi: 10.1016/j.bja.2018.06.030. Epub 2018 Sep 6. Br J Anaesth. 2018. PMID: 30442260 Free article.
Childhood Langerhans cell histiocytosis with severe lung involvement: a nationwide cohort study.
Le Louet S, Barkaoui MA, Miron J, Galambrun C, Aladjidi N, Chastagner P, Kebaili K, Armari-Alla C, Lambilliotte A, Lejeune J, Moshous D, Della Valle V, Sileo C, Ducou Le Pointe H, Chateil JF, Renolleau S, Piloquet JE, Portefaix A, Epaud R, Chiron R, Bugnet E, Lorillon G, Tazi A, Emile JF, Donadieu J, Héritier S. Le Louet S, et al. Among authors: portefaix a. Orphanet J Rare Dis. 2020 Sep 9;15(1):241. doi: 10.1186/s13023-020-01495-5. Orphanet J Rare Dis. 2020. PMID: 32907615 Free PMC article.
Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study.
Cances C, Vlodavets D, Comi GP, Masson R, Mazurkiewicz-Bełdzińska M, Saito K, Zanoteli E, Dodman A, El-Khairi M, Gorni K, Gravestock I, Hoffart J, Scalco RS, Darras BT; ANCHOVY Working Group. Cances C, et al. Orphanet J Rare Dis. 2022 Jul 29;17(1):300. doi: 10.1186/s13023-022-02455-x. Orphanet J Rare Dis. 2022. PMID: 35906608 Free PMC article.
40 results