Bremova-Ertl, Tatiana http://orcid.org/0000-0002-7397-045X
Claassen, Jens
Foltan, Tomas
Gascon-Bayarri, Jordi
Gissen, Paul
Hahn, Andreas
Hassan, Anhar
Hennig, Anita
Jones, Simon A.
Kolnikova, Miriam
Martakis, Kyriakos
Raethjen, Jan
Ramaswami, Uma
Sharma, Reena
Schneider, Susanne A.
Clinical trials referenced in this document:
Documents that mention this clinical trial
Efficacy and safety of N-acetyl-l-leucine in Niemann–Pick disease type C
https://doi.org/10.1007/s00415-021-10717-0
Funding for this research was provided by:
IntraBio Ltd.
Universität Bern
Article History
Received: 6 May 2021
Revised: 12 July 2021
Accepted: 13 July 2021
First Online: 13 August 2021
Declarations
:
: TBE received honoraria for lecturing from Actelion and Sanofi Genzyme and fees for the blinded rater services from IntraBio. JC and JR received fees for the blinded rater services from IntraBio. AH has received previous support from AbbVie and NCATS, and serves on the Editorial Board of Parkinsonism and Related Disorders. UR has received research grants from Amicus and Takeda, advisory board and lecture fee from Amicus, Sanofi Genzyme, and Takeda; all other authors declare no competing interests.
: This study was conducted in accordance with the International Conference for Harmonisation (of Technical Requirements for Pharmaceuticals for Human Use)—Good Clinical Practice Guideline, the General Data Protection Regulator, and the Declaration of Helsinki. Approval was obtained by the applicable responsible central research ethics committees / institutional review boards for each center (Ethics Committee of Ludwig Maximilian University of Munich (19-135), Slovakia: National Institute of Child Diseases Bratislava Ethics Committee (EudraCT 2018-004431-71), Bellvitge Hospital University Clinical Research Ethics Committee (AC001/19), East Midlands—Derby Research Ethics Committee (259038), Mayo Clinic Institutional Review Board (18-011974)).
: Written informed consent was obtained for all study participants by the subject or, if applicable, their parent or legal representative.
: All authors had full access to all the data in the study and had final responsibility for the decision to submit for publication. The study documents related to the study, and datasets generated and analyzed during the current study are not publicly available. No individual, de-identified participant data will be shared.