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 ...

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., ...

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.

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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 ...

By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against cancer cells in specific patient groups, according to a new study. The ...

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 ...