United for TSC
2025 International TSC Research Meeting: The picture of collaboration The 2025 International TSC (Tuberous Sclerosis Complex) Research Conference, held June 26-28 in Bethesda, MD, was well attended by the UTHealth Houston TSC Center of Excellence (TSC CoE) care team. “We…
A Noble Journey: Grateful Patient Gives Back to Help Others with Rare Diseases
Rylee Noble often watched other kids do cartwheels on the playground, wondering why she couldn’t do the same. She didn’t know that the joint stiffness and muscle aches she felt weren’t normal. “When you don’t know anything else, you don’t…
Two decades of spina bifida research published in Science
The culmination of over two decades of research from Hope Northrup, MD, professor, and Kit Sing Au, PhD, associate professor in the Department of Pediatrics, on the genetic factors associated with spina bifida has been published in Science. Kit Sing Au, PhD According to…
Clinical drug trial research brings hope for children who have rare tumor disease
Cheryl Navar was 24 weeks into her pregnancy when doctors found multiple tumors in her unborn baby’s heart. Before baby Nora entered the world, her soon-to-be parents sat in their hometown in Corpus Christi to learn about a rare disease…
Second patient in the world receives Moderna mRNA trial infusion at UTHealth Houston for glycogen storage disease
Imagine having to drink a solution of cornstarch and water every four hours to survive, and that missing a dose, even by 15 minutes, could lead to seizures and death. This is the reality for Rebecca Tarrence, who has glycogen…