Duan, Dongsheng http://orcid.org/0000-0003-4109-1132
Goemans, Nathalie
Takeda, Shin’ichi http://orcid.org/0000-0003-4780-6290
Mercuri, Eugenio
Aartsma-Rus, Annemieke http://orcid.org/0000-0003-1565-654X
Article History
Accepted: 22 January 2021
First Online: 18 February 2021
Competing interests
: D.D. is a member of the scientific advisory board for Solid Biosciences and an equity holder of Solid Biosciences. D.D. is an inventor on patents licensed to various companies. D.D. has served as an ad hoc consultant for 4DMT, Decibel Therapeutics, Evox, Primary Insight, Vida Ventures, Global Guidepoint and GLG consultancy in the past 3 years. The lab of D.D. has received research support from Solid Biosciences and Edgewise Therapeutics in the past 3 years. N.G. has received compensation as member of scientific boards or as speaker at symposia from Sarepta, Pfizer, Italpharmaco and PTC Therapeutics. S.T. has patents on sequences for exon skip by antisense nucleic acids as a member of NCNP together with Nippon Shinyaku. As principal inventor of these patents, S.T. is entitled to a share of royalties. S.T. discloses being an ad hoc consultant for Ono Pharmaceutical, Daiichisankyo, Asahikasei Pharma, Teijin Pharma, AGADA Biosciences, and Wave and being a member of the scientific advisory boards of Nippon Shinyaku, Taiho Pharma and Sarepta therapeutics. S.T. received speaker honoraria from Japan Health Science Foundation and Astellas Pharma and has also received research supports from Taiho Pharma, Daiichisankyo, Nippon Shinyaku, Takeda Pharmaceutical and the Noguchi Institute in the past 3 years. E.M. is a Principal Investigator in clinical trials and advisory board member for Sarepta, Santhera, PTC, Roche, Italfarmaco, NS Pharma and Pfizer. A.A.-R. discloses being employed by Leiden University Medical Center (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. is entitled to a share of royalties. A.A.-R. further discloses being an ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, CRISPR Therapeutics, Summit PLC, Alpha Anomeric, BioMarin Pharmaceuticals Inc., Eisai, Astra Zeneca, Santhera, Audentes, Global Guidepoint and GLG consultancy, Grunenthal, Wave, and BioClinica, having been a member of the Duchenne Network Steering Committee (BioMarin), and being a member of the scientific advisory boards of ProQR, Hybridize Therapeutics, Silence Therapeutics, Sarepta Therapeutics and Philae Pharmaceuticals. Remuneration for these activities is paid to LUMC. LUMC also received speaker honoraria from PTC Therapeutics and BioMarin Pharmaceuticals and funding for contract research from Italpharmaco and Alpha Anomeric. Project funding is received from Sarepta Therapeutics.
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