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

The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...

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 ...

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

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.

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

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 ...

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.

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

Reuters, the news and media division of Thomson Reuters, is the world’s largest multimedia news provider, reaching billions of people worldwide every day. Reuters provides business, financial ...

More than 40,000 Gen Z and millennial Bumble users worldwide, including over 2,000 singles in India, participated in the survey.