A Summerville man became the first person in South Carolina to undergo potentially life-changing gene therapy.

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

The heterogeneous nature of DBA mutations makes developing a single therapy challenging. Moreover, current gene therapy ...

This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.

The therapy, developed by PTC Therapeutics, is designed for patients across the severity of spectrum of AADC deficiency. It ...