Fields, T http://orcid.org/0000-0001-9506-4055
M. Bremova, T
Billington, I
Churchill, GC
Evans, W
Fields, C
Galione, A
Kay, R
Mathieson, T
Martakis, K
Patterson, M
Platt, F
Factor, M
Strupp, M
Funding for this research was provided by:
IntraBio Ltd
Article History
Received: 29 September 2022
Accepted: 22 May 2023
First Online: 29 May 2023
Declarations
:
: The study has been approved by the responsible central research ethics committees/institutional review boards, including the Ethics Committee of Ludwig Maximilian University of Munich (21–1269), National Institute of Child Diseases Bratislava Ethics Committee (EudraCT 2021–005356-10), Ethics Committee of General University Hospital in Prague (56/22 S-MEK), East Midlands – Derby Research Ethics Committee (1004498), Amsterdam UMC Locatie AMC Central Ethics Committee (NL79787.018.21), Mayo Clinic Institutional Review Board (22–001734), Emory University Institutional Review Board (STUDY00003227) Switzerland Business Administration System for Ethics Committees (2022–00638), and Victoria Human Research Ethics Committee (HREC/86167/MH-2022).Informed consent will be obtained for all study participants by the principal investigator/delegated investigator.
: Not applicable.
: MF is a co-founder, shareholder, and chairman of IntraBio. TF and IB are officers and shareholders of IntraBio. CF Is an employee and shareholder of IntraBio. FP is a cofounder, shareholder, and consultant to IntraBio and consultant to Actelion. MS is a shareholder to IntraBio and consultant for Abbott, Actelion, AurisMedical, Heel, IntraBio, Sensorion, and Vertify; he has received speaker’s honoraria from Abbott, Actelion, Auris Medical, Biogen, Eisai, Grünenthal, GSK, Henning Pharma, Interacoustics, Johnson & Johnson, MSD, Otometrics, Pierre-Fabre, TEVA, and UCB. AG and GC are cofounders, shareholder sand consultants to IntraBio. MP is a shareholder of IntraBio and has received consulting fees, honoraria, and research grants from Actelion Pharmaceuticals Ltd. and Biomarin. RK is a consultant to IntraBio. TBE received honoraria for lecturing from Actelion and Sanofi Genzyme and fees for the blinded rater services from IntraBio.IntraBio owns patents EP3359146, EP3416631, EP3482754, USPTO10905670, and USPTO11400067 (application 16/324,301) related to treatment of lysosomal storage disorders and neurodegenerative diseases with acetyl-leucine and its analogs.IntraBio has pending patent applications EP19174007.5, EP20215877.0, PCT/IB2017/054928, PCT/IB2017/054929, PCT/US2018/056420, PCT/IB2018/054676, PCT/US2018/018420, PCT/IB2019/060525, PCT/IB2019/051214, PCT/GB2017/051090, PCT/IB2020/051767, PCT/IB2020/056096, and PCT/IB2021/050236 relating to treatment of lysosomal storage disorders, neurodegenerative diseases, and neurodegeneration with acetyl-leucine and its analogs.