Article History
Received: 21 January 2026
Accepted: 31 May 2026
First Online: 16 July 2026
Declarations
Ethics approval and consent to participate: Not applicable.
Consent for publication: Not applicable.
Competing interests: JAB receives research support from Avrobio, BioMarin Pharmaceutical, Chiesi Farmaceutici, Denali Therapeutics, Idorsia Pharmaceuticals, Pfizer, Protalix BioTherapeutics, Sangamo Therapeutics, Sanofi, Takeda, and Travere Therapeutics, and has participated in advisory boards for Chiesi and Mirum Pharmaceuticals. PA receives grant/research support from Takeda, and speaker/travel honoraria from Alexion, Amicus Therapeutics, BioMarin, Chiesi, Sanofi-Genzyme, Takeda, and Ultragenyx. DH has received honoraria for speaking and consulting fees for advisory boards from AceLinkAcelink, Amicus, Chiesi, Freeline, Uniqure, Sangamo, Sanofi, and Takeda, administered through University College London consultants, and used in part to support research in lysosomal storage diseases.