Sandra had the opportunity to participate in the RARE Drug Development Symposium as a scholarship recipient and represent the G1DF. It was a wonderful opportunity to learn about different approaches other rare disease organizations are taking to develop treatments for their diseases, in addition to networking with other advocates, researchers and industry.

This was the 8th meeting organized by Global Genes and the Orphan Disease Center at the University of Pennsylvania and took place in Philadelphia from May 1st-3rd. Participants in this symposium included rare disease patients, patient advocate groups, parents, researchers working on rare diseases, pharma, biotech companies and government agencies. The purpose of this meeting is to equip advocates with knowledge, skills and connections needed to advance therapy development in rare disease communities.

Sandra was also invited to participate in the poster session with findings from our Collective Voices in Glut1 Deficiency Project, highlighting patient experiences and priorities as we look for better and easier ways to diagnose and treat this disease (see the poster here).

The meeting featured different panels with diverse guest speakers including fellow patient advocacy group members as well as leaders from the FDA and NIH. The panelists discussed topics such as the path to treatments, understanding data for basic research, new technologies in early stage research, the regulatory landscape, and research readiness. Also, there were workshops in which attendees could interact with the panelists in smaller groups and ask questions. 

One of the highlights of the meeting was the chance to meet one-on-one with experts in different rare disease areas and ask direct questions and guidance about our path and the role that rare disease organizations like ours play in the development of better treatments for our patient community.