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156 results

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Page 1
The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.
Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C, Fischer D, Gerlach I, Heinig K, Bugawan T, Dziadek S, Kinch R, Czech C, Khwaja O. Kletzl H, et al. Among authors: marquet a. Neuromuscul Disord. 2019 Jan;29(1):21-29. doi: 10.1016/j.nmd.2018.10.001. Epub 2018 Oct 30. Neuromuscul Disord. 2019. PMID: 30553700 Clinical Trial.
A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier.
Sturm S, Günther A, Jaber B, Jordan P, Al Kotbi N, Parkar N, Cleary Y, Frances N, Bergauer T, Heinig K, Kletzl H, Marquet A, Ratni H, Poirier A, Müller L, Czech C, Khwaja O. Sturm S, et al. Among authors: marquet a. Br J Clin Pharmacol. 2019 Jan;85(1):181-193. doi: 10.1111/bcp.13786. Epub 2018 Nov 16. Br J Clin Pharmacol. 2019. PMID: 30302786 Free PMC article. Clinical Trial.
Longitudinal characterization of biomarkers for spinal muscular atrophy.
Bonati U, Holiga Š, Hellbach N, Risterucci C, Bergauer T, Tang W, Hafner P, Thoeni A, Bieri O, Gerlach I, Marquet A, Khwaja O, Sambataro F, Bertolino A, Dukart J, Fischmann A, Fischer D, Czech C. Bonati U, et al. Among authors: marquet a. Ann Clin Transl Neurol. 2017 Apr 11;4(5):292-304. doi: 10.1002/acn3.406. eCollection 2017 May. Ann Clin Transl Neurol. 2017. PMID: 28491897 Free PMC article.
Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M, Jablonski P, Jacobsen B, Khwaja O, Kletzl H, Ko CP, Kustermann S, Marquet A, Metzger F, Mueller B, Naryshkin NA, Paushkin SV, Pinard E, Poirier A, Reutlinger M, Weetall M, Zeller A, Zhao X, Mueller L. Ratni H, et al. Among authors: marquet a. J Med Chem. 2018 Aug 9;61(15):6501-6517. doi: 10.1021/acs.jmedchem.8b00741. Epub 2018 Jul 25. J Med Chem. 2018. PMID: 30044619
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
Outcome of patients with large B-cell lymphoma treated with tafasitamab plus lenalidomide either before or after CAR T-cell therapy.
Camus V, Houot R, Brisou G, Tessoulin B, Bailly S, Sesques P, Decroocq J, Krzisch D, Oberic L, Lemonnier F, Bouabdallah K, Campidelli A, Tounes L, Abraham J, Herbaux C, Morschhauser F, Damaj GL, Guidez S, Carras S, Fornecker LM, Choquet S, Hermine O, Paillassa J, Chauchet A, Casasnovas O, Drieu La Rochelle L, Castilla-Llorente C, Joris M, Dupont V, Marquet A, Le Gouill S, Jardin F. Camus V, et al. Among authors: marquet a. Blood Adv. 2024 Oct 22;8(20):5371-5381. doi: 10.1182/bloodadvances.2024013726. Blood Adv. 2024. PMID: 39163620 Free PMC article.
Author Correction: Quantum control of a cat qubit with bit-flip times exceeding ten seconds.
Réglade U, Bocquet A, Gautier R, Cohen J, Marquet A, Albertinale E, Pankratova N, Hallén M, Rautschke F, Sellem LA, Rouchon P, Sarlette A, Mirrahimi M, Campagne-Ibarcq P, Lescanne R, Jezouin S, Leghtas Z. Réglade U, et al. Among authors: marquet a. Nature. 2024 Jun;630(8015):E1. doi: 10.1038/s41586-024-07565-z. Nature. 2024. PMID: 38760464 No abstract available.
156 results