Creation of NC Rare Disease Advisory Council Provides Unique Opportunities

“The creation of the Rare Disease Advisory Council represents a unique opportunity for the UNC School of Medicine and others in North Carolina to serve the people of the state and beyond,” said Bruce Cairns, director of the North Carolina Jaycee Burn Center at UNC-Chapel Hill. “We strongly believe a partnership between advocacy groups including Taylor’s Tale, academic medical centers, the National Institutes of Health and others keenly interested in the diagnosis and treatment of rare diseases can be a model for the nation as we tackle some of the most challenging diseases affecting our citizens. We are grateful that the General Assembly has passed the bill and that Governor McCrory has now signed it. We are ready to get to work.” 

Rare Disease Day Symposium on Collaborating to Impact Patient Lives: Highlights and Summary

The Symposium, held Feb. 24 at Syneos Health in Morrisville, NC, brought together clinical researchers in industry and academia, advocates, patients and their families, health care providers, legislators and sponsors to discuss “the present and future state of clinical research in rare disease and the latest initiatives driving change.”

Rare Disease Day

For Rare Disease Day, a conversation with a family with a rare disease.

NC Rare Disease Advisory Council Associate Chair represented on steering committee of The Forum for Collaborative Research focuses on Rare Disease

Tara Britt, Associate Chair of the NC Rare Disease Advisory Council, has joined the steering committee of The Forum for Collaborative Research’s Rare Diseases Forum which facilities drug development for the treatment of inborn errors of metabolism and other genetically based rare diseases.

3 New rare diseases added to NC Newborn Screening panel

A provision in the NC budget announced this week adds three tests to the state’s newborn screening panel. The new screenings will test for Pompe disease, Mucopolysaccharidosis type 1 (MDS 1), and X-linked Adrenoleukodystropy (X-ALD).