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

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

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

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

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

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

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

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

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

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

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 ...