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Gene-Targeted Therapies for Rare Diseases: Early Diagnosis and Equitable Delivery

Thursday, June 17, 2021 9:00am to 1:30pm

Virtual Event Free Event
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This Virtual NIH Roundtable meeting, comprised of three half-day sessions spanning three weeks in June, is co-hosted by NCATS, the Eunice Kennedy Shriver NICHD, and NINDS.

Thursdays, June 3, 10, 17, 9:00 a.m. to 1:30 p.m. PDT

Registration is required and is free.

Audience: The meeting is calibrated to be of interest to professionals as well as lay people, parents, affected individuals, and advocates.

Description: Scientists, clinicians, therapeutics developers, patient representatives, economists, newborn screening experts, and ethicists will discuss challenges and barriers to equitable and timely delivery of gene-targeted therapies to individuals living with rare diseases and will identify possible solutions for these issues.

The goals for this meeting are to:

  • Explore new and existing approaches to identify all individuals with rare diseases that would benefit from gene-targeted therapies.
  • Identify existing systems that can be leveraged or adapted to bring treatments to the individuals who can benefit from them.
  • Identify gaps that need to be addressed and solutions to facilitate the effective dissemination of treatments in an equitable manner.

 

Website | Agenda | Registration | Speakers

Attendees are required to register prior to joining. The videocast link will be emailed to registrants prior to the event.

 

Tippi MacKenzie, MD, UCSF Professor of Surgery and Co-director of the Center for Maternal-Fetal Precision Medicine is a co-chair for this meeting.

Event Details