Pipeline

Knowledge-intensive network with integrated drug development expertise

Publications


Emcitate

van Geest FS et al.  Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study, The Journal of Clinical Endocrinology & Metabolism, Volume 107, Issue 3, March 2022, Pages e1136–e1147, https://doi.org/10.1210/clinem/dgab750

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, et al. Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study.Lancet Diabetes Endocrinol. 2020;8(7):594-605.

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, et al. 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. Lancet Diabetes Endocrinol. 2019;7(9):695-706.

 

Aladote

Morrison EE, Oatey K, Gallagher B, Grahamslaw J, O’Brien R, Black P, Oosthuyzen W, Lee RJ, Weir CJ, Henriksen D, Dear JW; POP Trial Investigators. Principal results of a randomised open label exploratory, safety and tolerability study with calmangafodipir in patients treated with a 12 h regimen of N-acetylcysteine for paracetamol overdose (POP trial). EBioMedicine. 2019;46:423-430.

POP Trial Investigators, Dear J. Randomised open label exploratory, safety and tolerability study with calmangafodipir in patients treated with the 12-h regimen of N-acetylcysteine for paracetamol overdose-the PP100-01 for Overdose of Paracetamol (POP) trial: study protocol for a randomised controlled trial. Trials. 2019;20(1):27.

 

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