Gene therapy pioneer Jude Samulski, PhD, professor of pharmacology at the UNC School of Medicine, is featured in this NPR story about a 30-year research journey from idea to treatment reality for kids with muscular dystrophy.
UNC School of Medicine scientists led by Aravind Asokan, PhD, reveal how certain gene-carrying AAV vectors can penetrate the brain more efficiently to treat brain and spinal cord conditions, while reducing liver payload.
Approach developed in part by UNC’s Dr. Steven Gray restored enzyme function and extended survival time in treated mice.
Submitted by Taylor’s Tale North Carolina’s Leadership Enhances National Rare Disease Initiatives When Governor McCrory signed the legislation enacting the North Carolina Advisory Council on Rare Diseases on August 5, 2015, the state claimed a leadership role by creating the first such council in the country. By passing the legislation unanimously, the General Assembly … Read more
Contact: Taylor Holgate (Burr) 202-228-1616 Wednesday April 6, 2016 WASHINGTON – Today, Senator Burr (R-NC) spoke before the Health, Education, Labor and Pensions (HELP) Committee on the need for rare disease treatments. WATCH: Sen. Burr speaks on treatments for rare diseases Senator Burr highlighted the example of Taylor King from Charlotte, NC who suffers … Read more
Laura King Edwards will run the Martha’s Vineyard 20 Miler Road Race in honor of her younger sister Taylor.
Triangle Business Journal on Monday announced the 21 winners for its of the 2016 Health Care Heroes Awards.
Sharon King Daniel S. Levine, producer of RARECast on Global Genes RARE Daily, interviews Sharon King, rare disease advocate and president of Taylor’s Tale, and Tara Britt, associate director of the newly created North Carolina Rare Disease Advisory Council, about the development of the legislation that created the advisory council on rare diseases in North … Read more
Dr. Bruce Cairns, John Stackhouse Distinguished Professor of Surgery and Director of the North Carolina Jaycee Burn Center in the UNC School of Medicine, serves as Chair of the Council.
On October 16, 2015, CBS Evening News aired a story on Lori Sames’ search for a cure for her daughter, Hannah, who suffers from Giant Axonal Neuropathy disease (GAN), a rare disease which causes nerves to die and muscles to stop working. Lori and her husband, Matt, founded Hannah’s Hopeto raise money to fund the … Read more