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Authors
Hipp, Jörg F. https://orcid.org/0000-0002-7875-2988
Bacino, Carlos A.
Bird, Lynne M. https://orcid.org/0000-0003-4833-3747
Bruenig-Traebert, Ina
Chan, Daniel
de Wit, Marie Claire https://orcid.org/0000-0002-9042-4813
Fontoura, Paulo
Hooper, Gregory
Jagasia, Ravi
Krishnan, Michelle L.
Murtagh, Lorraine
Noci, Alessandro
Martínez, Ana Roche https://orcid.org/0000-0001-8295-1982
Schwab, Dietmar
Serrano, Mercedes https://orcid.org/0000-0002-2056-2428
Shen, Mark D. https://orcid.org/0000-0002-1190-7981
Tillmann, Julian
Tjeertes, Jorrit
Vincenzi, Brenda
Berry-Kravis, Elizabeth https://orcid.org/0000-0001-7099-1522
Bonni, Azad
,
Barakovic, Muhamed
Bon, Charlotte
Boonsimma, Ponghatai
Bustamante, Mariana
Buzasi, Katalin
Chamberlain, Stormy
Clinch, Susanne
Connor-Ahmad, Siobhan
Daetwyler, Philipp
Danila, Oana
Holst, Sebastian
Kremer, Thomas
Lipsmeier, Florian
Magon, Stefano
Messer, Amanda
Nobbs, David
Palermo, Giuseppe
Squassante, Lisa
Wiese, Thomas
,
Bain, Jennifer M.
Capal, Jamie
Gavrilova, Ralitza
Cabeza, María Muñoz
Pietrafusa, Nicola
Rajamaran, Rajsekar
Jeste, Shafali
Vigevano, Federico
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Article History
Received: 13 January 2025
Accepted: 19 May 2025
First Online: 11 July 2025
Competing interests
: J.F.H., D.C., G.H., L.M., A.N., D.S., J. Tillmann and A.B. are employees of F. Hoffmann-La Roche. I.B.T., M.L.K., J. Tjeertes, B.V., P.F. and R.J. are former employees of F. Hoffmann-La Roche. M.L.K. is currently an employee of Biomedical Research, Translational Medicine, Novartis AG, Basel, Switzerland. J. Tjeertes is currently an employee of Bayer AG, Basel Switzerland. P.F. is Chief Scientific and Clinical Development Advisor, Stalicla SA, Geneva, Switzerland. L.M.B. has received funding from Aardvark Therapeutics, Acadia Pharmaceuticals, Biogen, Ionis Pharmaceuticals, F. Hoffman-La Roche, Gedeon Richter, Neuren Pharmaceuticals, Ovid Therapeutics, PTC Therapeutics, Radius Health, Soleno Therapeutics and Ultragenyx to conduct clinical trials or consult on trial design or results. E.B.-K. has received funding from Acadia, Alcobra, AMO, Asuragen, Avexis, Biogen, BioMarin, Cydan, Engrail, Erydel, Fulcrum, GeneTx, GW, Healx, Ionis, Jaguar, Kisbee, Lumos, Marinus, Mazhi, Moment Biosciences, Neuren, Neurogene, Neurotrope, Novartis, Orphazyme/Kempharm/Zevra, Ovid, PTC Therapeutics, Retrophin, Roche, Seaside Therapeutics, Taysha, Tetra, Ultragenyx, Yamo, Zynerba and Vtesse/Sucampo/Mallinckrodt Pharmaceuticals, to consult on trial design or run clinical or lab validation trials in genetic neurodevelopmental or neurodegenerative disorders, all of which are directed to RUMC in support of rare disease programs. E.B.-K. receives no personal funds, and Rush University Medical Center has no relevant financial interest in any of the commercial entities listed. For M.C.d.W., Erasmus MC received compensation for participation in the Tangelo trial from Hoffmann-La Roche, and for consultation from Jazz Pharmaceuticals. M.D.S. has received research funding from Biogen, Ionis and Roche to conduct clinical trials or research studies. All funds were paid to the University of North Carolina; M.D.S. receives no personal funds and has no relevant financial interest in any of the commercial entities listed. The other authors declare no competing interests.
