Trace Neuroscience, which launched this week with $101 million, benefited from last year’s approval of Qalsody. Its CEO spoke ...

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

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

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

PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...

To determine the type and severity of a cancer, pathologists typically analyze thin slices of a tumor biopsy under a microscope.

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

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

Tel Aviv University researchers have made a discovery that could enhance our understanding of genetic mutations and their ...

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 ...

At the BioFuture 2024 conference held in New York in November, Seema Kumar, the CEO of Cure, described women’s health as something that has been directed at the “bikini area.” That “bikini” bias ...