Currently, technological developments are offering the hope of new therapies for individuals with rare disease at a pace that has the potential for significantly challenging the existing infrastructure for efficient, effective, and equitable delivery. It is important therefore, to plan and be prepared for these challenges. Changes need to be implemented to improve the process of getting more treatments to more patients, more quickly.

June 3rd will focus on defining:

• Who are the individuals that could benefit from gene-targeted therapies – now and in the future? 

• What novel approaches are needed to enable development of gene-targeted therapies for all genetic rare diseases – now and in the future?

• When is the optimal time to identify individuals who could benefit from gene-targeted therapies (e.g., newborn screening)