Rare Diseases

The Gulf Coast Cluster for Rare Diseases (GCCl RD) will be the first centralized resource for the rare disease community in the Texas Medical Center. It will focus on advancing orphan drug development, diagnostics, clinical trial resources, patient education, and advocacy for rare diseases. By bringing together faculty, students, postdocs, academics, industry partners, FDA and NIH experts, the cluster aims to foster collaboration and innovation in this understudied area.

A rare disease is defined by the Orphan Drug Act of 1983 as one affecting fewer than 200,000 people. With over 7,000 identified rare diseases (including Huntingdon’s, ALS), around 300 million people worldwide are affected. Most rare diseases are genetic, often presenting in childhood, and 95% lack approved treatments.

The collaborative group under GCCl RD aims to unite all GCC members, providing a centralized resource to drive scientific research, clinical management, diagnostics, and the pursuit of multi-institutional grants specifically focused on rare disease research and translation.  This initiative leverages the strengths of multiple institutions to tackle challenges in rare disease research more collectively and effectively.

Opportunities for engagement include:

• Workshops in collaboration with the National Organization for Rare Diseases (NORD)
• Sessions hosted by the NIH National Center for Advancing Translational Sciences
• Educating researchers on funding opportunities to foster innovative projects
• Involvement of trainees in direct interactions with rare disease foundations and patients to enrich their educational experience and nurture future leaders in the field.
• Annual events, such as Rare Disease Day and the GCCl RD Conference to bring together global experts to discuss advancements and promote networking, furthering collective knowledge and support.

Chair: Thomas Kent, MD, Texas A&M University, Institute of Biosciences and Technology

Co-Chair: Kenneth Ramos, MD, PhD, Texas A&M University, Institute of Biosciences and Technology

Co-Chair:  Laura Lavery, PhD, Rice University