Kuseyri Hübschmann, Oya http://orcid.org/0000-0001-6576-6566
Horvath, Gabriella
Cortès-Saladelafont, Elisenda
Yıldız, Yılmaz http://orcid.org/0000-0001-9076-1388
Mastrangelo, Mario
Pons, Roser
Friedman, Jennifer
Mercimek-Andrews, Saadet http://orcid.org/0000-0001-8396-6764
Wong, Suet-Na
Pearson, Toni S. http://orcid.org/0000-0002-3679-7707
Zafeiriou, Dimitrios I.
Kulhánek, Jan
Kurian, Manju A.
López-Laso, Eduardo
Oppebøen, Mari
Kılavuz, Sebile
Wassenberg, Tessa
Goez, Helly
Scholl-Bürgi, Sabine
Porta, Francesco
Honzík, Tomáš
Santer, René
Burlina, Alberto
Sivri, H. Serap http://orcid.org/0000-0001-8260-9984
Leuzzi, Vincenzo
Hoffmann, Georg F.
Jeltsch, Kathrin
Hübschmann, Daniel
Garbade, Sven F. http://orcid.org/0000-0002-7420-4757
Assmann, Birgit
Fung, Cheuk-Wing
Guder, Philipp
Hong, Stacey Tay Kiat
Karall, Daniela
Kato, Mitsuhiro
Kavecan, Ivana
Koht, Jeanette Aimee
Kuster, Alice
Lücke, Thomas
Manti, Filippo
Mir, Pablo
Mühlhausen, Chris
Önenli Mungan, Halise Neslihan
Palacios, Natalia Alexandra Julia
Ramos, Joaquín Alejandro Fernández
Steel, Dora
Stevanović, Galina
Sykut-Cegielska, Jolanta
Verbeek, Marcel M.
García-Cazorla, Angeles
Opladen, Thomas http://orcid.org/0000-0003-4349-7662
,
Funding for this research was provided by:
Dietmar Hopp Stiftung
Rosetrees Trust
Article History
Received: 9 December 2020
Accepted: 12 August 2021
First Online: 20 September 2021
Competing interests
: A.G.C. receives teaching honorarium from PTC Therapeutics GT, Inc. C.M. and E.L.L. have received consultancy honorarium as part of the Advisory Board of PTC Therapeutics GT, Inc. G.F.H. receives teaching as well as consultancy honorarium from PTC Therapeutics GT, Inc. J.F. had trials with Biogen (Angelman’s Syndrome) and Stealth Biotherapeutics (Mitochondrial Disorders); J.F.’s spouse is Founder and Principal of Friedman Bioventure, which holds a variety of publicly traded and private biotechnology interests. O.K.H. and T.W. have received teaching honorarium from PTC Therapeutics GT, Inc. R.P. has received honoraria as a speaker from Genesis Pharma, PTC Therapeutics GT, Inc. and as consultant in Advisory board of PTC Therapeutics GT, Inc. T.O. receives teaching honorarium and research support from PTC Therapeutics GT, Inc. V.L. has received consultancy honoraria as part of Advisory Boards organized by PTC Therapeutics GT, Inc., BioMarin Pharmaceutical Inc. and Homology Medicines. The remaining authors declare no competing interests.