1. Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy. Issue 2 (27th December 2019) Authors: Signorelli, Mirko; Ayoglu, Burcu; Johansson, Camilla; Lochmüller, Hanns; Straub, Volker; Muntoni, Francesco; Niks, Erik; Tsonaka, Roula; Persson, Anja; Aartsma‐Rus, Annemieke; Nilsson, Peter; Al‐Khalili Szigyarto, Cristina; Spitali, Pietro Journal: Journal of cachexia, sarcopenia and muscle Issue: Volume 11:Issue 2(2020) Page Start: 505 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. PO195 Outcome measures for duchenne muscular dystrophy: implications for trials. (1st December 2017) Authors: Domingos, Joana; Eagle, Michelle; Moraux, Amelie; Butler, Jordan; Decostre, Valerie; Ridout, Deborah; Mayhew, Anna; Selby, Victoria; Guglieri, Michela; Holst, Menno Van der; Jansen, Merel; Verschuuren, Jan GM; Groot, Imelda de; Niks, Erik; Servais, Laurent; Hogrel, Jean-Yves; Straub, Volker; Voit... Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 88(2017)Supplement 1 Page Start: A63 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Issue 8 (July 2016) Authors: Straub, Volker; Balabanov, Pavel; Bushby, Kate; Ensini, Monica; Goemans, Nathalie; De Luca, Annamaria; Pereda, Alejandra; Hemmings, Robert; Campion, Giles; Kaye, Edward; Arechavala-Gomeza, Virginia; Goyenvalle, Aurelie; Niks, Erik; Veldhuizen, Olav; Furlong, Pat; Stoyanova-Beninska, Violeta; Wood... Journal: Lancet neurology Issue: Volume 15:Issue 8(2016:Jul.) Page Start: 882 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗