Healthcare utilization and disease burden in children with metachromatic leukodystrophy in Germany
Diagnostic and treatment routine pathways and sociomedical support in MLD require extensive resources. We provide detailed cross-sectional and long-term data of MLD-associated disease burden in different stages of disease. This data may serve as a reference when analyzing disease- and healthcare burden also after gene-/stem cell-therapy.
Authors
- Christa Raabe
- Pascal Martin (ORCID: https://orcid.org/0000-0002-0678-6417)
- Gregor Starc (ORCID: https://orcid.org/0000-0001-9822-0308)
- Samuel Groeschel (ORCID: https://orcid.org/0000-0002-2706-7163)
- Ingeborg Krägeloh‐Mann (ORCID: https://orcid.org/0000-0003-4599-1373)
- Christiane Kehrer (ORCID: https://orcid.org/0000-0002-6313-1839)
- Andrea Bevot (ORCID: https://orcid.org/0000-0002-9482-9447)
Institutions
- University Children's Hospital Tübingen (DE)
- Hertie Institute for Clinical Brain Research (DE)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-05-23
- DOI
- https://doi.org/10.1186/s13023-025-03637-z
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Deutsche Forschungsgemeinschaft
- Bundesministerium für Bildung und Forschung
- Universitätsklinikum Tübingen