One multi-stakeholder and multi-purpose registry
The MLD initiative collects patient data for multiple purposes. One of the rationales of the registry is to avoid fragmentation of registries for MLD. In rare diseases, international clustering of patients is essential to create reliable study populations. Therefore, multi-purpose and multi-stakeholder registries are important.
Purposes
The collected data in the MLDi can be used for:
❶ Academic research, e.g. to investigate genotype-phenotype correlations, natural history, and prognostic factors.
❷ Postmarketing surveillance after approval of new treatments.
❸ Health-technology assessments to evaluate the social, economic, organisational, and ethical issues of health interventions.
Contribute to the MLD initiative
As a clinician or researcher you can contribute to the MLD registry. More information can be found here.
Use the data
You can do a data request by following the research policy of the MLD initiative.
