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

Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new tab gene therapy to ...

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

At Neurogene, the developer of a gene therapy for Rett syndrome, expediency seems to be the priority, @adamfeuerstein writes.

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 for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...

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

The FDA has accepted for review the resubmitted BLA for prademagene zamikeracel for recessive dystrophic epidermolysis bullosa.

Understanding what to patent versus what to keep as a trade secret is an increasingly important and challenging decision ...

University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...