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Authors
Hammond, Suzan M https://orcid.org/0000-0002-5640-7569
Aartsma‐Rus, Annemieke https://orcid.org/0000-0003-1565-654X
Alves, Sandra https://orcid.org/0000-0002-8881-9197
Borgos, Sven E https://orcid.org/0000-0002-6222-9252
Buijsen, Ronald A M https://orcid.org/0000-0002-9722-8110
Collin, Rob W J https://orcid.org/0000-0003-4347-6503
Covello, Giuseppina https://orcid.org/0000-0001-5660-8283
Denti, Michela A https://orcid.org/0000-0001-7203-7062
Desviat, Lourdes R https://orcid.org/0000-0002-2081-0815
Echevarría, Lucía https://orcid.org/0000-0001-9307-6992
Foged, Camilla https://orcid.org/0000-0003-2812-5588
Gaina, Gisela https://orcid.org/0000-0001-7415-5281
Garanto, Alejandro https://orcid.org/0000-0001-5721-1560
Goyenvalle, Aurelie T https://orcid.org/0000-0003-3938-1165
Guzowska, Magdalena
Holodnuka, Irina https://orcid.org/0000-0002-7208-4499
Jones, David R
Krause, Sabine https://orcid.org/0000-0002-3141-886X
Lehto, Taavi https://orcid.org/0000-0002-7131-2998
Montolio, Marisol https://orcid.org/0000-0001-5494-5737
Van Roon‐Mom, Willeke https://orcid.org/0000-0002-3035-0533
Arechavala‐Gomeza, Virginia https://orcid.org/0000-0001-7703-3255
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Funding
Funding for this research was provided by:
European Cooperation in Science and Technology (CA17103)
MEC | Instituto de Salud Carlos III (CPII17/00004)
EC | European Regional Development Fund (CPII17/00004)
Ikerbasque, Basque Foundation for Science
UKRI | Medical Research Council (MR/R025312/1)
Duchenne Parent Project
Stichting Spieren voor Spieren (SvS15‐06)
Prinses Beatrix Spierfonds (SvS15‐06)
Duchenne UK
EC | Horizon 2020 Framework Programme (Project BIND)
Algemene Nederlandse Vereniging ter voorkoming van Blindheid (UZ2015‐31)
Stichting Blinden‐Penning (UZ2015‐31)
Landelijke Stichting voor Blinden en Slechtzienden (UZ2015‐31)
Landelijke Stichting voor Blinden en Slechtzienden (UZ2018‐31)
Stichting Oogfonds Nederland (UZ2015‐31)
Stichting Oogfonds Nederland (UZ2018‐31)
Stichting Macula Degeneratie Fonds (UZ2015‐31)
Stichting Macula Degeneratie Fonds (UZ2018‐31)
Stichting Retina Nederland Fonds (UZ2015‐31)
Rotterdamse Stichting Blindenbelangen (UZ2015‐31)
Rotterdamse Stichting Blindenbelangen (UZ2018‐31)
Stichting Blindenhulp (UZ2015‐31)
Stichting Blindenhulp (UZ2018‐31)
Stichting tot Verbetering van het Lot der Blinden (UZ2015‐31)
Stichting voor Ooglijders (UZ2015‐31)
Stichting Dowilvo (UZ2015‐31)
Stichting Dowilvo (UZ2018‐31)
Foundation Fighting Blindness USA (PPA‐0517‐0717‐RAD)
Duchenne Parent Project Spain
Hersenstichting (DR‐2018‐00253)
Ministry of Research and Innovation in Romania/National Program (31N/2016/PN 16.22.02.05)
Fundação para a Ciência e a Tecnologia‐MCTES, Portugal (PTDC/BBB‐BMD/6301/2014)
Fundación Ramón Areces (Grant XVII CN)
Spanish Ministry of Science and Innovation (PID2019‐105344RB‐I00)
Eesti Teadusagentuur (grant PSG226)
Friedrich‐Baur‐Stiftung
Danish Council for Independent Research, Technology and Production Sciences (DFF‐4184‐00422)
ZonMw Programme Translational Research 2 (446002002)
Campaign Team Huntington
French Muscular Dystrophy Association (Project number 20577)
EC | Horizon 2020 Framework Programme (B‐SMART 721058)
EC | Horizon 2020 Framework Programme (REFINE 761104)
Institut National de la Santé et de la Recherche Médicale
Association Monegasque contre les myopathies
Muscular Dystrophy UK (19GRO‐PG36‐0294)
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Article History
Received: 6 August 2020
Revised: 27 November 2020
Accepted: 30 November 2020
First Online: 6 April 2021
Conflict of interest
: A.A‐R discloses being employed by LUMC which has patents on exon skipping technology. As co‐inventor of some of these patents, AAR is entitled to a share of royalties. AAR further discloses being ad hoc consultant for PTC Therapeutics, Sarepta Therapeutics, Eisa Pharmaceuticals, WaVe Life Sciences, Alpha Anomeric, CRISPR Therapeutics, BioMarin Pharmaceuticals Inc., Global Guidepoint and GLG consultancy, Grunenthal and BioClinica, being a member of the Duchenne Network Steering Committee (BioMarin) and of the scientific advisory boards of ProQR and Philae Pharmaceuticals. Remuneration for these activities is paid to LUMC. LUMC also received speaker honoraria from PTC Therapeutics and BioMarin Pharmaceuticals. A.G and R.W.J.C are inventors of several patents describing the use of antisense oligonucleotides for the treatment of inherited retinal diseases. C.F is ad hoc consultant for Lundbeck Pharma A/S, Valby, DK. W.vR.M discloses being employed by LUMC which has patents on exon skipping technology for brain disorders. As co‐inventor of some of these patents WvRM is entitled to a share of royalties. T.L is a consultant for and has equity interests in Evox Therapeutics Ltd., Oxford, UK. L.E is an employee of SQY therapeutics developing tcDNA antisense oligonucleotides. M.A.D and G.C are co‐inventors of patent WO2016/151523 (RNA interference mediated therapy for neurodegenerative diseases) filed by the University of Trento and are entitled to a share of royalties. S.M.H is an inventor on a patent describing cell‐penetrating peptides and is employed by Oxford Biomedica Plc, Oxford UK. Co‐authors, R.A.M.B, G.G, S.A, L.R.D, S.K, V.A‐G, S.E.B and A.T.G declare no conflict of interests.
