UTSW scientists treat rare genetic disease with designer molecule
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with…
What families should know about the genetic disorder
Imagine facing a condition so rare that fewer than 100 people in the world are known to have…
North Texas girl to become first patient to receive treatment for rare genetic disorder
An 11-year-old from Rowlett is set to make medical history as the first person in the world to…