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Authors
Schuele, Rebecca
Synofzik, Matthis https://orcid.org/0000-0002-2280-7273
Graessner, Holm
Aartsma-Rus, Annemieke https://orcid.org/0000-0003-1565-654X
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Article History
First Online: 13 September 2024
Competing interests
: R.S., M.S., H.G. and A.A.R. are coordinators of the 1M1M initiative. H.G. and A.A.R. are board of directors members and R.S., M.S. and A.A.R. are members of the scientific advisory committee of N1C. A.A.R. discloses employment by LUMC, which has patents on exon-skipping technology, some of which has been licensed to BioMarin and subsequently sublicensed to Sarepta. As co-inventor of some of these patents, A.A.R. was entitled to a share of royalties. A.A.R. further discloses serving as ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, Regenxbio, Dyne Therapeutics, Lilly, BioMarin Pharmaceuticals Inc., Eisai, Entrada, Takeda, Splicesense, Galapagos, Sapreme, Italfarmaco and Astra Zeneca. In the past 5 years, A.A.R. also performed ad hoc consulting for Alpha Anomeric. A.A.R. also reports membership of the scientific advisory boards of Eisai, Hybridize Therapeutics, Silence Therapeutics, Sarepta Therapeutics, Sapreme and Mitorx. In the past 5 years, A.A.R. was also a scientific advisory board member for ProQR. Remuneration for A.A.R.’s consulting and advising activities is paid to LUMC. In the past 5 years, LUMC also received speaker honoraria from PTC Therapeutics, Alnylam Netherlands, Italfarmaco and Pfizer and funding for contract research from Sapreme, Eisai, Galapagos, Synaffix and Alpha Anomeric. Project funding is received from Sarepta Therapeutics and Entrada via unrestricted grants. H.G. has nothing to disclose in relation to the topics covered in this manuscript. In the past 5 years, he has also received consultancy honoraria from UCB. M.S. received consultancy honoraria from Ionis, UCB, Prevail, Orphazyme, Biogen, Servier, Reata, GenOrph, AviadoBio, Biohaven, Zevra, Lilly and Solaxa in the past 5 years, all unrelated to the present manuscript. R.S. has nothing to disclose in relation to the topics covered in this manuscript. She has received speaker and/or consultancy honoraria or sponsoring contributions from Abbvie, Bial, STADA and Everpharma in the past 5 years.
