The Critical Role Registries Play In Rare Diseases

Rare diseases with single gene mutations are now showing great potential for treatment and, in some cases, even the potential for a cure. Certain cancers are also being treated using these techniques. New products are rapidly being introduced to the marketplace, and the number of early stage assets are expanding rapidly. Several different treatment approaches are used, but all strategies aim to target the correction of a mutated or missing gene.

UBC's Dr. Don Gabriel, Medical Oncology & Scientific Strategy and Colleen Valenzuela, Clinical Operations share their insights on the importance of registries in rare diseases.