August 15, 2018
(Dallas Morning News) - A McKinney family and UT Southwestern scientists race to save boy with ultra-rare disease
Ever since doctors diagnosed his son with a rare disease, Doug Woleben has spent every evening on his computer, searching for a way to save the 6-year-old's life. He has mastered the foreign language of medical literature, corresponded with drug companies, flown his family long distances for experimental treatments and emailed academic researchers who might know of something, anything, that could help his son. Some days have brought hope, but many more have brought frustration. Read the Full Story
Just as the Wolebens were running low on options, their neurologist at UT Southwestern Medical Center sent Will's case file to a new colleague named Steven Gray. Gray, a scientist who joined the faculty in December, has made it his mission to develop treatments for fatal childhood diseases like Will's. Not only would this approach, known as custom gene therapy, target Will's symptoms, it might fix their underlying cause: a single defective gene out of Will's more than 20,000. Read the full story (Dallas Morning News).
Additional stories in the Dallas Morning News about Will Woleben:
September 13, 2018 - Anonymous donor gives $155,000 to help McKinney boy with rare disease
November 15, 2018 - McKinney boy's story moves Mark Cuban to invest in research targeting ultra-rare, fatal disease
Gene therapy center developing treatments for more than two dozen rare neurological diseases largely overlooked by the medical field.
New facility addresses national shortage of a clinical-grade virus, which can be loaded with genes and delivered into human cells.
Video: Engineering hope: UT Southwestern targets rare diseases
"Our work here is changing medicine, said Dr. Steven Gray, Associate Professor of Pediatrics, Molecular Biology, and Neurology & Neurotherapeutics, who organized Chrissy's clinical trial with the National Institutes of Health and has pioneered gene therapy treatments for other rare diseases. "The need is so great. We meet with the families and see the effects of these horribles diseases. It goes through our minds every day."