Key lessons from the first international treatment eligibility committee: the case of metachromatic leukodystrophy

Approved

Classifications

MinEdu publication type
A1 Journal article (peer-reviewed)
Definition
Article
Target group
Scientific
Peer reviewed
Peer-reviewed
Article type
Journal article
Host publication type
Journal

Authors of the publication

Number of authors
39
Authors
Schoenmakers, Daphne H; Asbreuk, Marije A B C; Martin, Tamara; Datema, Mareen; Beerepoot, Shanice; Inbar-Feigenberg, Michal; Groeschel, Samuel; Kehrer, Christiane; Øberg, Andreas; Sevin, Caroline; Fumagalli, Francesca; Bergner, Caroline G; Vieira, Päivi; Bley, Annette; Uusimaa, Johanna; Horn, Morten Andreas; Brožová, Klára; Stögmann, Eva; Pichler, Herbert; Lüftinger, Roswitha; Eklund, Erik A; Mochel, Fanny; Adang, Laura A; Laugwitz, Lucia; Boelens, Jaap Jan; Calbi, Valeria; Darling, Alejandra; García-Cazorla, Ángeles; Grønborg, Sabine W; Lindemans, Caroline A; van Hasselt, Peter M; Hollak, Carla E M; de Koning, Tom J; Ram, Dipak; Dekker, Hanka; Schöls, Ludger; Zerem, Ayelet; Graessner, Holm; Wolf, Nicole I

Publication channel information

Title of journal/series
European journal of paediatric neurology
ISSN (print)
1090-3798
ISSN (electronic)
1532-2130
ISSN (linking)
1090-3798
Publisher
Elsevier
Publication forum ID
55773
Publication forum level
1
Country of publication
United Kingdom
Internationality
Yes

Detailed publication information

Publication year
2025
Reporting year
2025
Journal/series volume number
57
Page numbers
72-81
DOI
10.1016/j.ejpn.2025.05.012
Language of publication
English

Co-publication information

International co-publication
Yes
Co-publication with a company
No

Availability

Link to self-archived version
https://urn.fi/URN:NBN:fi:oulu-202506094235

Classification and additional information

MinEdu field of science classification
3124 Neurology and psychiatry, 3121 General medicine, internal medicine and other clinical medicine

Funding information

Funding information in the publication
This work is generated within the European Reference Network for Rare Neurological Diseases, funded by the European Union and as part of the MLD initiative funded by the Dutch Healthcare Institute (“Zorginstituut Nederland”) and the platform Medicine for Society (“Medicijn voor de Maatschappij”).

Source database ID

WoS ID
WOS:001509210500002
Scopus ID
2-s2.0-105007432940
Other database ID
PMID: 40482356