It’s a tradition that whenever a breakthrough is made in treating children with genetic disorders, Laura Case, PhD, PT, DPT, MS, PCS, C/NDT, gathers the Duke Doctor of Physical Therapy students and they celebrate with cake.
Recently, they celebrated three exciting breakthroughs in the past months:
- The approval in December of two gene therapy treatments for sickle cell disease
- The first gene therapy dosing, in January, of a child with Rett syndrome, in a clinical trial
- The approval in March of a drug for Duchenne muscular dystrophy that may help to activate muscle repair mechanisms to increase muscle fiber regeneration, reduce inflammation, and reduce fibrosis
Dr. Case said, “The celebration is not only to take joy in each new discovery that will change children’s lives for the better, but also to remember that anything is possible and that we will always believe in, encourage, and support our students in their quest to strive for the best.”
