Document is current

Article History

Received: 19 December 2024

Accepted: 27 October 2025

First Online: 5 December 2025

Declarations

:

: Not applicable.

: Not applicable.

: CL is an employee of F. Hoffmann-La Roche Ltd. AK is an employee of ZS Associates India Pvt Ltd. AT is an employee of ZS Associates International Inc. UO is an employee of F. Hoffmann-La Roche Ltd. MP has been on advisory boards for Novartis Gene Therapies, Roche, Biogen and PTC and has received speaking fees/honoraria from Novartis Gene Therapies, Pfizer, Roche, Biogen and PTC. VS has been on advisory boards for Astellas Gene Therapies, Biogen, Edgewise Therapeutics, Ipsen, Kate Therapeutics, ML Bio Solutions, Novartis Gene Therapies, PepGen, Roche, Sanofi, Sarepta Therapeutics, Vertex Pharmaceuticals and Wave Therapeutics, has received speaking fees/honoraria from Novartis Gene Therapies, Pfizer, Roche, Sanofi and Sarepta Therapeutics and has research collaborations with Sarepta Therapeutics and Sanofi. MCW has participated in scientific advisory boards and reports receiving funding for travel or speaker honoraria or consultancies from Affinia, Argenyx, Aro, Audentes Therapeutics, AveXis Pharma, Biogen Pharma GmbH, BridgeBio, Colloquium Neurologicum, Edgewise, Fulcrum Therapeutics, GLG Consult, Grünenthal Pharma AG, Guidepoint Global, ML Bio, Neurix, Novartis Pharma GmbH, Pfizer Inc., PharNEXT, PTC Therapeutics, Roche Pharma AG, RTI Health Solutions, Santhera Pharmaceuticals, Sarepta Therapeutics Inc., Teva, Ultragenyx and Wave Sciences Technology and has received research support from Gamedis Study, GlaxoSmithKline GmbH, Grifols GmbH, Novartis Pharma GmbH, PharNEXT and Trophos AG (now Roche Pharma AG).