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Page 1
Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study.
Groeneweg S, van Geest FS, Abacı A, Alcantud A, Ambegaonkar GP, Armour CM, Bakhtiani P, Barca D, Bertini ES, van Beynum IM, Brunetti-Pierri N, Bugiani M, Cappa M, Cappuccio G, Castellotti B, Castiglioni C, Chatterjee K, de Coo IFM, Coutant R, Craiu D, Crock P, DeGoede C, Demir K, Dica A, Dimitri P, Dolcetta-Capuzzo A, Dremmen MHG, Dubey R, Enderli A, Fairchild J, Gallichan J, George B, Gevers EF, Hackenberg A, Halász Z, Heinrich B, Huynh T, Kłosowska A, van der Knaap MS, van der Knoop MM, Konrad D, Koolen DA, Krude H, Lawson-Yuen A, Lebl J, Linder-Lucht M, Lorea CF, Lourenço CM, Lunsing RJ, Lyons G, Malikova J, Mancilla EE, McGowan A, Mericq V, Lora FM, Moran C, Müller KE, Oliver-Petit I, Paone L, Paul PG, Polak M, Porta F, Poswar FO, Reinauer C, Rozenkova K, Menevse TS, Simm P, Simon A, Singh Y, Spada M, van der Spek J, Stals MAM, Stoupa A, Subramanian GM, Tonduti D, Turan S, den Uil CA, Vanderniet J, van der Walt A, Wémeau JL, Wierzba J, de Wit MY, Wolf NI, Wurm M, Zibordi F, Zung A, Zwaveling-Soonawala N, Visser WE. Groeneweg S, et al. Among authors: van der spek j, van geest fs, van der knaap ms, van der knoop mm, van der walt a, van beynum im. Lancet Diabetes Endocrinol. 2020 Jul;8(7):594-605. doi: 10.1016/S2213-8587(20)30153-4. Lancet Diabetes Endocrinol. 2020. PMID: 32559475 Free PMC article.
Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial.
Groeneweg S, Peeters RP, Moran C, Stoupa A, Auriol F, Tonduti D, Dica A, Paone L, Rozenkova K, Malikova J, van der Walt A, de Coo IFM, McGowan A, Lyons G, Aarsen FK, Barca D, van Beynum IM, van der Knoop MM, Jansen J, Manshande M, Lunsing RJ, Nowak S, den Uil CA, Zillikens MC, Visser FE, Vrijmoeth P, de Wit MCY, Wolf NI, Zandstra A, Ambegaonkar G, Singh Y, de Rijke YB, Medici M, Bertini ES, Depoorter S, Lebl J, Cappa M, De Meirleir L, Krude H, Craiu D, Zibordi F, Oliver Petit I, Polak M, Chatterjee K, Visser TJ, Visser WE. Groeneweg S, et al. Among authors: van der walt a, van der knoop mm, van beynum im. Lancet Diabetes Endocrinol. 2019 Sep;7(9):695-706. doi: 10.1016/S2213-8587(19)30155-X. Epub 2019 Jul 31. Lancet Diabetes Endocrinol. 2019. PMID: 31377265 Free PMC article. Clinical Trial.
Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study.
van Geest FS, Groeneweg S, van den Akker ELT, Bacos I, Barca D, van den Berg SAA, Bertini E, Brunner D, Brunetti-Pierri N, Cappa M, Cappuccio G, Chatterjee K, Chesover AD, Christian P, Coutant R, Craiu D, Crock P, Dewey C, Dica A, Dimitri P, Dubey R, Enderli A, Fairchild J, Gallichan J, Garibaldi LR, George B, Hackenberg A, Heinrich B, Huynh T, Kłosowska A, Lawson-Yuen A, Linder-Lucht M, Lyons G, Monti Lora F, Moran C, Müller KE, Paone L, Paul PG, Polak M, Porta F, Reinauer C, de Rijke YB, Seckold R, Menevşe TS, Simm P, Simon A, Spada M, Stoupa A, Szeifert L, Tonduti D, van Toor H, Turan S, Vanderniet J, de Waart M, van der Wal R, van der Walt A, van Wermeskerken AM, Wierzba J, Zibordi F, Zung A, Peeters RP, Visser WE. van Geest FS, et al. Among authors: van toor h, van wermeskerken am, van den akker elt, van der walt a, van der wal r, van den berg saa. J Clin Endocrinol Metab. 2022 Feb 17;107(3):e1136-e1147. doi: 10.1210/clinem/dgab750. J Clin Endocrinol Metab. 2022. PMID: 34679181 Free PMC article.
Anti-inflammatory disease-modifying treatment and disability progression in primary progressive multiple sclerosis: a cohort study.
Lorscheider J, Kuhle J, Izquierdo G, Lugaresi A, Havrdova E, Horakova D, Hupperts R, Duquette P, Girard M, Prat A, Grand'Maison F, Grammond P, Sola P, Ferraro D, Trojano M, Ramo-Tello C, Lechner-Scott J, Pucci E, Solaro C, Slee M, Van Pesch V, Sanchez Menoyo JL, van der Walt A, Butzkueven H, Kappos L, Kalincik T; MSBase Study Group. Lorscheider J, et al. Among authors: van der walt a, van pesch v. Eur J Neurol. 2019 Feb;26(2):363-370. doi: 10.1111/ene.13824. Epub 2018 Nov 2. Eur J Neurol. 2019. PMID: 30298572 Free article.
Serum phosphorylated neurofilament-heavy chain levels in multiple sclerosis patients.
Gresle MM, Liu Y, Dagley LF, Haartsen J, Pearson F, Purcell AW, Laverick L, Petzold A, Lucas RM, Van der Walt A, Prime H, Morris DR, Taylor BV; Ausimmune Consortium; Shaw G, Butzkueven H. Gresle MM, et al. Among authors: van der walt a. J Neurol Neurosurg Psychiatry. 2014 Nov;85(11):1209-13. doi: 10.1136/jnnp-2013-306789. Epub 2014 Mar 17. J Neurol Neurosurg Psychiatry. 2014. PMID: 24639436
Comparative Effectiveness and Cost-Effectiveness of Natalizumab and Fingolimod in Patients with Inadequate Response to Disease-Modifying Therapies in Relapsing-Remitting Multiple Sclerosis in the United Kingdom.
Spelman T, Herring WL, Zhang Y, Tempest M, Pearson I, Freudensprung U, Acosta C, Dort T, Hyde R, Havrdova E, Horakova D, Trojano M, De Luca G, Lugaresi A, Izquierdo G, Grammond P, Duquette P, Alroughani R, Pucci E, Granella F, Lechner-Scott J, Sola P, Ferraro D, Grand'Maison F, Terzi M, Rozsa C, Boz C, Hupperts R, Van Pesch V, Oreja-Guevara C, van der Walt A, Jokubaitis VG, Kalincik T, Butzkueven H; MSBase Investigators. Spelman T, et al. Among authors: van der walt a, van pesch v. Pharmacoeconomics. 2022 Mar;40(3):323-339. doi: 10.1007/s40273-021-01106-6. Epub 2021 Dec 18. Pharmacoeconomics. 2022. PMID: 34921350 Free PMC article.
Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis.
Lefort M, Sharmin S, Andersen JB, Vukusic S, Casey R, Debouverie M, Edan G, Ciron J, Ruet A, De Sèze J, Maillart E, Zephir H, Labauge P, Defer G, Lebrun-Frenay C, Moreau T, Berger E, Clavelou P, Pelletier J, Stankoff B, Gout O, Thouvenot E, Heinzlef O, Al-Khedr A, Bourre B, Casez O, Cabre P, Montcuquet A, Wahab A, Camdessanché JP, Maurousset A, Ben Nasr H, Hankiewicz K, Pottier C, Maubeuge N, Dimitri-Boulos D, Nifle C, Laplaud DA, Horakova D, Havrdova EK, Alroughani R, Izquierdo G, Eichau S, Ozakbas S, Patti F, Onofrj M, Lugaresi A, Terzi M, Grammond P, Grand'Maison F, Yamout B, Prat A, Girard M, Duquette P, Boz C, Trojano M, McCombe P, Slee M, Lechner-Scott J, Turkoglu R, Sola P, Ferraro D, Granella F, Shaygannejad V, Prevost J, Maimone D, Skibina O, Buzzard K, Van der Walt A, Karabudak R, Van Wijmeersch B, Csepany T, Spitaleri D, Vucic S, Koch-Henriksen N, Sellebjerg F, Soerensen PS, Hilt Christensen CC, Rasmussen PV, Jensen MB, Frederiksen JL, Bramow S, Mathiesen HK, Schreiber KI, Butzkueven H, Magyari M, Kalincik T, Leray E. Lefort M, et al. Among authors: van wijmeersch b, van der walt a. BMC Med Res Methodol. 2022 May 30;22(1):155. doi: 10.1186/s12874-022-01623-8. BMC Med Res Methodol. 2022. PMID: 35637426 Free PMC article.
203 results