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Page 1
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
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.
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.
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
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.
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 May 6. doi: 10.1038/s41586-024-07294-3. Online ahead of print. Nature. 2024. PMID: 38710932
The use of copper isotopes for understanding metal transfer mechanisms within the continuum mine-river-dam (Huelva Region, Spain).
Viers J, Freydier R, Grande JA, Zouiten C, Marquet A, Delpoux S, Santisteban M, Pokrovsky OS, Fortes JC, Davila JM, Sarmiento A, Audry S, Luis A, Meheut M, Behra P, Darrozes J, Monnin C. Viers J, et al. Among authors: marquet a. Environ Sci Pollut Res Int. 2023 Apr;30(18):53275-53294. doi: 10.1007/s11356-023-25802-2. Epub 2023 Feb 28. Environ Sci Pollut Res Int. 2023. PMID: 36853539
153 results