Bringing the Gorlin Syndrome Community into the Rare Disease Conversation

GSA Natural History Study Director Jean Pickford and Executive Director Meredith Weiss joined more than 1,000 attendees at the National Organization for Rare Disorders (NORD) Breakthrough Summit in Washington, D.C., October 25 to 27. Speakers included NIH Director Dr. Jay Bhattacharya and Acting FDA Commissioner Kyle Diamantas, bringing federal research and regulatory leadership into discussions about the future of rare disease care and treatment development.

For GSA, the meeting offered an opportunity to work with NORD on strengthening our Natural History Study through its IAMRARE platform. Continuing to improve this study helps us make participation more accessible and build data that researchers can use to better understand Gorlin syndrome.

We also connected with other rare disease organizations to exchange practices and ideas for growing our impact, reach and engaging our communities in research. Discussions about biotech investment trends and the regulatory climate provided context for the opportunities and challenges facing rare disease treatment development.