Schoenmakers, Daphne H.
Mochel, Fanny
Adang, Laura A.
Boelens, Jaap-Jan
Calbi, Valeria
Eklund, Erik A.
Grønborg, Sabine W.
Fumagalli, Francesca
Groeschel, Samuel
Lindemans, Caroline
Sevin, Caroline
Schöls, Ludger
Ram, Dipak
Zerem, Ayelet
Graessner, Holm
Wolf, Nicole I. http://orcid.org/0000-0003-1721-0728
Article History
Received: 14 November 2023
Accepted: 3 February 2024
First Online: 7 February 2024
Declarations
:
: The authors confirm that they have read the Journal's position on issues involved in ethical publication and affirm that this report is consistent with those guidelines. All participants gave permission for their survey responses to be used.
: Not applicable.
: CS is advisor and/or investigator for clinical trials in Metachromatic Leukodystrophy and other leukodystrophies (Bluebird Bio, Shire/Takeda, Minoryx Therapeutics, Orchard, Ionis). FF, VC are investigators of hematopoietic stem cell gene therapy clinical trials for MLD sponsored by Orchard Therapeutics, the license holder of investigational medicinal product OTL-200. FF and VC act as consultant for ad hoc Advisory board of Orchard Therapeutics. HG is coordinator of ERN-RND and University Hospital Tübingen has received funding from the EU for network coordination and management. EAE has participated in Advisory Boards for Orchard Therapeutics. JB is consultant for Omeros, Bluerock, Sanofi, Sobi, Advanced Clinical, Bluebird Bio, SmartImmune and Immusoft. LAA is consultant for Takeda and Orchard Therapeutics. LS is consultant for Vico Therapeutics, participated in a Novartis advisory board and is investigator for Vigil Neuroscience unrelated to MLD and HSCT. NIW is advisor and/or co-investigator for clinical trials in Metachromatic Leukodystrophy and other leukodystrophies (Shire/Takeda, Orchard, Ionis, PassageBio, VigilNeuro, Sana Biotech, Lilly), without personal payment. SWG and CL have participated in an Orchard Therapeutics advisory board. SG was supported by DFG grant GR 4688/2-1 and received institutional research support from Takeda and Orchard. He is advisor and coinvestigator for trials in MLD (Takeda, Orchard) but receives no personal payment related to this role. The other authors (AZ, DHS, DR, FM) have nothing to declare. The following authors are part of the MLD initiative: DHS, FM, LAA, JJB, VC, EAE, SWG, FF, SG,CL, CS, LS, DR, AZ, and NIW.