Document is current

Article History

Received: 27 April 2020

Accepted: 28 December 2020

First Online: 22 January 2021

Ethics approval and consent to participate

: The studies have been approved by the responsible central research ethics committees/institutional review boards, including the Ethics Committee of Ludwig Maximilian University of Munich (19-135; 19-119; 20-079), National Institute of Child Diseases Bratislava Ethics Committee (EudraCT 2018-004431-71), Bellvitge Hospital University Clinical Research Ethics Committee (AC001/19; AC004/19), Hospital University La Paz (5621); East Midlands – Derby Research Ethics Committee (259038), North West- Greater Manchester South Research Ethics Committee (260774), Office for Research Ethics Committees Northern Ireland (260363), Mayo Clinic Institutional Review Board (18-011974; 19-000373), Office of Science and Research Institutional Review Board, New York University School of Medicine (i17-01666), and University of California Los Angeles Institutional Review Board (19-000348; 19-000442).Informed consent will be obtained for all study participants.

: Not applicable.

: MF is a co-founder, shareholder, and Chairman of IntraBio. TF and IB are officers and shareholders of IntraBio. FP is a cofounder, shareholder, and consultant to IntraBio and consultant to Actelion and Orphazyme. MS is a shareholder to IntraBio, and consultant for Abbott, Actelion, AurisMedical, Heel, IntraBio and Sensorion; 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, UCB. He is the distributor of M-glasses and positional vertigo App. AG and GC are cofounders, shareholders, and consultants to IntraBio. MP is a shareholder of IntraBio and has received consulting fees, honoraria, and research grants from Actelion Pharmaceuticals Ltd. and Biomarin. CV and SF are consultants and shareholders of IntraBio. UG, TM, GB, and RKay are consultants to IntraBio. TBE received honoraria for lecturing from Actelion and Sanofi Genzyme.IntraBio owns patents EP3359146 and EP3416631 (related to treatment of lysosomal storage disorders and neurodegenerative diseases with acetyl-leucine and its analogues).IntraBio has pending patent applications EP19174007.5, EP3482754, PCT/US2018/056420, PCT/US2018/018420, PCT/IB2018/054676, PCT/IB2019/051214, PCT/IB2017/054928, PCT/GB2017/051090, PCT/IB2017/054929, USPTO 62/812,987, USPTO 62/842,296, USPTO 62/888,894, USPTO 62/895,144, USPTO 62/868,383, USPTO 62/931,003, USPTO 62/960,637, and PCT/IB2019/060525 relating to treatment of lysosomal storage disorders, neurodegenerative diseases, and neurodegeneration with acetyl-leucine and its analogues.