Patient Registries
INFORM RARE is contributing to the co-design, enhancement, and implementation of Canadian patient registries.
Registry Values
Metabolic Patient Registries
Registries currently in development
The Canadian MPS Registry, in partnership with the Canadian MPS Society
The Canadian PKU Registry, in partnership with Canadian PKU & Allied Disorders Inc.
Objectives
Furthering the understanding of the natural history of these rare inherited metabolic diseases;
Evaluating intervention effectiveness (e.g., through registry-based randomized trials);
Providing a clinical contact database for academic- and industry-sponsored research; and
Allowing patient organizations and their partners to promote and support patient-oriented research (research that focuses on the priorities of patients and their caregivers).
Governance
SMA Registry
Please visit the INFORM SMA webpage for more information.