||NIH invites stakeholders throughout the scientific research, advocacy, clinical practice, industry, and lay communities, including the general public, to attend the 3-day meeting and submit comments or questions relating to the effective, efficient and equitable distribution of gene-targeted therapies for individuals with rare diseases.
The goal of Day 1 is to discuss:
For more information on the meeting and to view the agenda for all three days, visit: https://events-support.com/events/Gene-Targeted_Therapies_June_2021
- 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)?