Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

Immunotherapy, which enhances the immune system's T cell response to eliminate cancer cells, has emerged as a key approach in ...

Why one trial participant's healthy birth after her CAR-T treatment for lupus is catching resesarchers' attention: ...

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

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

By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups.

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult ...

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 involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

As a person who “reads all the therapy books,” she said, “I haven’t really seen it be wrong.” Anxious, depressed or just lonely, people who can’t find or afford a professional ...

As a person who “reads all the therapy books,” she said, “I haven’t really seen it be wrong.” Anxious, depressed or just lonely, people who can’t find or afford a professional ...