Funding for this research was provided by:
Wellcome Trust (202834/Z/16/Z)
Action Medical Research (xxx)
Text and Data Mining valid from 2018-08-16
Received: 5 July 2018
Accepted: 29 July 2018
First Online: 16 August 2018
Ethics approval and consent to participate
: Standard protocol approvals, registrations and patient consents relate to this study.Research on data obtained from NPC patients were covered by REC/IRB approvals 06/MRE02/85 (UK) and S-032/2012 (Germany). Written informed consent, and if applicable, assent, were obtained in each centre.All study participants and/or guardians of patients gave their informed consent to participation in the compassionate use of ADLL. This was a compassionate use observational study.
: Not applicable.
: FMP is a cofounder and consultant to IntraBio; and consultant to Actelion and Orphazyme. RL has received honoraria and travel support from Actelion and CTD Holdings. PF, MS and FP and DtV are shareholder in IntraBio. HR is a full-time employee at Biogen Inc. MS is Joint Chief Editor of the Journal of Neurology, Editor in Chief of Frontiers of Neuro-otology and Section Editor of F1000. He has received speaker’s honoraria from Abbott, Actelion, Auris Medical, Biogen, Eisai, Grünenthal, GSK, Henning Pharma, Interacoustics, MSD, Otometrics, Pierre-Fabre, TEVA, UCB. He acts as a consultant for Abbott, Actelion, AurisMedical, Heel, IntraBio and Sensorion. TBE received travel grants and speaker’s honoraria from Actelion and Sanofi-Genzyme. EM has received speaker’s and consultation honoraria from Actelion, Biomarin, Orphazyme, Sanofi-Genzyme and Shire.
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