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Journal Abstract Search

295 related items for PubMed ID: 34800400

  • 21. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.
    Schoser B, Stewart A, Kanters S, Hamed A, Jansen J, Chan K, Karamouzian M, Toscano A.
    J Neurol; 2017 Apr; 264(4):621-630. PubMed ID: 27372449
    [Abstract] [Full Text] [Related]

  • 22. Pirfenidone in patients with unclassifiable progressive fibrosing interstitial lung disease: a double-blind, randomised, placebo-controlled, phase 2 trial.
    Maher TM, Corte TJ, Fischer A, Kreuter M, Lederer DJ, Molina-Molina M, Axmann J, Kirchgaessler KU, Samara K, Gilberg F, Cottin V.
    Lancet Respir Med; 2020 Feb; 8(2):147-157. PubMed ID: 31578169
    [Abstract] [Full Text] [Related]

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  • 24. Upadacitinib as induction and maintenance therapy for moderately to severely active ulcerative colitis: results from three phase 3, multicentre, double-blind, randomised trials.
    Danese S, Vermeire S, Zhou W, Pangan AL, Siffledeen J, Greenbloom S, Hébuterne X, D'Haens G, Nakase H, Panés J, Higgins PDR, Juillerat P, Lindsay JO, Loftus EV, Sandborn WJ, Reinisch W, Chen MH, Sanchez Gonzalez Y, Huang B, Xie W, Liu J, Weinreich MA, Panaccione R.
    Lancet; 2022 Jun 04; 399(10341):2113-2128. PubMed ID: 35644166
    [Abstract] [Full Text] [Related]

  • 25. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.
    Case LE, Bjartmar C, Morgan C, Casey R, Charrow J, Clancy JP, Dasouki M, DeArmey S, Nedd K, Nevins M, Peters H, Phillips D, Spigelman Z, Tifft C, Kishnani PS.
    Neuromuscul Disord; 2015 Apr 04; 25(4):321-32. PubMed ID: 25617983
    [Abstract] [Full Text] [Related]

  • 26. Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease.
    Luquetti DV, Jeng LJB, Donohue KM, Maynard JW, Review TeamOffice of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA..
    J Inherit Metab Dis; 2024 Jul 04; 47(4):578-581. PubMed ID: 38768612
    [No Abstract] [Full Text] [Related]

  • 27. Simeprevir with pegylated interferon alfa 2a or 2b plus ribavirin in treatment-naive patients with chronic hepatitis C virus genotype 1 infection (QUEST-2): a randomised, double-blind, placebo-controlled phase 3 trial.
    Manns M, Marcellin P, Poordad F, de Araujo ES, Buti M, Horsmans Y, Janczewska E, Villamil F, Scott J, Peeters M, Lenz O, Ouwerkerk-Mahadevan S, De La Rosa G, Kalmeijer R, Sinha R, Beumont-Mauviel M.
    Lancet; 2014 Aug 02; 384(9941):414-26. PubMed ID: 24907224
    [Abstract] [Full Text] [Related]

  • 28. Simeprevir with pegylated interferon alfa 2a plus ribavirin in treatment-naive patients with chronic hepatitis C virus genotype 1 infection (QUEST-1): a phase 3, randomised, double-blind, placebo-controlled trial.
    Jacobson IM, Dore GJ, Foster GR, Fried MW, Radu M, Rafalsky VV, Moroz L, Craxi A, Peeters M, Lenz O, Ouwerkerk-Mahadevan S, De La Rosa G, Kalmeijer R, Scott J, Sinha R, Beumont-Mauviel M.
    Lancet; 2014 Aug 02; 384(9941):403-13. PubMed ID: 24907225
    [Abstract] [Full Text] [Related]

  • 29. Folic acid supplementation and malaria susceptibility and severity among people taking antifolate antimalarial drugs in endemic areas.
    Crider K, Williams J, Qi YP, Gutman J, Yeung L, Mai C, Finkelstain J, Mehta S, Pons-Duran C, Menéndez C, Moraleda C, Rogers L, Daniels K, Green P.
    Cochrane Database Syst Rev; 2022 Feb 01; 2(2022):. PubMed ID: 36321557
    [Abstract] [Full Text] [Related]

  • 30. Efficacy of mepolizumab add-on therapy on health-related quality of life and markers of asthma control in severe eosinophilic asthma (MUSCA): a randomised, double-blind, placebo-controlled, parallel-group, multicentre, phase 3b trial.
    Chupp GL, Bradford ES, Albers FC, Bratton DJ, Wang-Jairaj J, Nelsen LM, Trevor JL, Magnan A, Ten Brinke A.
    Lancet Respir Med; 2017 May 01; 5(5):390-400. PubMed ID: 28395936
    [Abstract] [Full Text] [Related]

  • 31. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
    McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E, Clinical Evaluator Training Group, ACT DMD Study Group.
    Lancet; 2017 Sep 23; 390(10101):1489-1498. PubMed ID: 28728956
    [Abstract] [Full Text] [Related]

  • 32. Efficacy and safety of suvratoxumab for prevention of Staphylococcus aureus ventilator-associated pneumonia (SAATELLITE): a multicentre, randomised, double-blind, placebo-controlled, parallel-group, phase 2 pilot trial.
    François B, Jafri HS, Chastre J, Sánchez-García M, Eggimann P, Dequin PF, Huberlant V, Viña Soria L, Boulain T, Bretonnière C, Pugin J, Trenado J, Hernandez Padilla AC, Ali O, Shoemaker K, Ren P, Coenjaerts FE, Ruzin A, Barraud O, Timbermont L, Lammens C, Pierre V, Wu Y, Vignaud J, Colbert S, Bellamy T, Esser MT, Dubovsky F, Bonten MJ, Goossens H, Laterre PF, COMBACTE Consortium and the SAATELLITE Study Group.
    Lancet Infect Dis; 2021 Sep 23; 21(9):1313-1323. PubMed ID: 33894131
    [Abstract] [Full Text] [Related]

  • 33. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec 23; 16(92):240-1. PubMed ID: 18092404
    [Abstract] [Full Text] [Related]

  • 34. The new horizons for treatment of Late-Onset Pompe Disease (LOPD).
    Guémy C, Laforêt P.
    Rev Neurol (Paris); 2023 Dec 23; 179(1-2):81-89. PubMed ID: 36609019
    [Abstract] [Full Text] [Related]

  • 35. Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.
    Berger KI, Chien YH, Dubrovsky A, Kishnani PS, Llerena JC, Neilan E, Roberts M, Sheng B, Batista JL, Periquet M, Wilson KM, van der Ploeg AT.
    J Neurol; 2024 Aug 23; 271(8):5433-5446. PubMed ID: 38896264
    [Abstract] [Full Text] [Related]

  • 36. Safety and efficacy of upadacitinib in combination with topical corticosteroids in adolescents and adults with moderate-to-severe atopic dermatitis (AD Up): results from a randomised, double-blind, placebo-controlled, phase 3 trial.
    Reich K, Teixeira HD, de Bruin-Weller M, Bieber T, Soong W, Kabashima K, Werfel T, Zeng J, Huang X, Hu X, Hendrickson BA, Ladizinski B, Chu AD, Silverberg JI.
    Lancet; 2021 Jun 05; 397(10290):2169-2181. PubMed ID: 34023009
    [Abstract] [Full Text] [Related]

  • 37. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.
    Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E.
    Lancet Haematol; 2021 May 05; 8(5):e323-e333. PubMed ID: 33838113
    [Abstract] [Full Text] [Related]

  • 38. Long-term follow-up from the ORATORIO trial of ocrelizumab for primary progressive multiple sclerosis: a post-hoc analysis from the ongoing open-label extension of the randomised, placebo-controlled, phase 3 trial.
    Wolinsky JS, Arnold DL, Brochet B, Hartung HP, Montalban X, Naismith RT, Manfrini M, Overell J, Koendgen H, Sauter A, Bennett I, Hubeaux S, Kappos L, Hauser SL.
    Lancet Neurol; 2020 Dec 05; 19(12):998-1009. PubMed ID: 33129442
    [Abstract] [Full Text] [Related]

  • 39. Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
    Tard C, Bouhour F, Michaud M, Beltran S, Fournier M, Demurger F, Lagrange E, Nollet S, Sacconi S, Noury JB, Magot A, Cintas P, Renard D, Deibener-Kaminsky J, Lefeuvre C, Davion JB, Salort-Campana E, Arrassi A, Taouagh N, Spinazzi M, Pompe Study Group, Attarian S, Laforêt P.
    Eur J Neurol; 2024 Jul 05; 31(7):e16292. PubMed ID: 38587143
    [Abstract] [Full Text] [Related]

  • 40. Improved efficacy of a next-generation ERT in murine Pompe disease.
    Xu S, Lun Y, Frascella M, Garcia A, Soska R, Nair A, Ponery AS, Schilling A, Feng J, Tuske S, Valle MCD, Martina JA, Ralston E, Gotschall R, Valenzano KJ, Puertollano R, Do HV, Raben N, Khanna R.
    JCI Insight; 2019 Mar 07; 4(5):. PubMed ID: 30843882
    [Abstract] [Full Text] [Related]

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