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Fernando Rosa, Liv Warfield, Theophilus London, Omar + QCBA, and Sister Nancy at the Camden venue.

Hunter syndrome is a rare disorder in which the body does not properly digest sugar molecules in the body.

Date Time Community Rallies to Raise $2.5 Million for Gene Therapy Trial at UNC The gene therapy trial has already received approval from the FDA for the investigational new drug application, so both UNC School of Medicine researchers and Project Alive are close to treating patients. Individuals with Hunter syndrome, a rare genetic condition with a variety of symptoms, are missing a working copy of the gene that codes for the enzyme Iduronate 2-Sulfatase (IDS), which is crucial for cells to remove waste from major organs such as the brain, heart, liver, and spleen. Without the enzyme, waste b...

TN-RDAC To Inform TennCARE On Pharmaceutical Treatments Wednesday, May 12, 2021 The newly formed Tennessee Rare Disease Advisory Council or TN-RDAC (pronounced AHR-dak) has set its priorities to advocate for Tennesseans diagnosed with rare diseases, including advising TennCare’s Pharmacy Advisory Committee and Drug Utilization Review Committee on pharmaceutical treatments, as well as identifying the overall impact of rare diseases on Tennesseans.   Created by public charter in the 111th Tennessee General Assembly, TN-RDAC elected at its inaugural meeting its Chairma...

 E-Mail Established in 2019 in Nashville, COMBINEDBrain is a non-profit consortium of patient advocacy groups, researchers and clinicians working together to fast-track cures and therapies for individuals afflicted with rare genetic neurodevelopmental disorders. Established in 2017, the Marlene and Spencer Hays Foundation has a mission to support efforts to improve peoples' lives through the arts; research and innovations in health care; education and programs to help at-risk youth; and to promote enhanced economic achievement by encouraging discipline, ambition and commitment to goals and ...