High cost is a major challenge. The global cell and gene therapy market is projected to expand by $6.56b, reflecting an ...

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.

A Summerville man became the first person in South Carolina to undergo potentially life-changing gene therapy.

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 can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

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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 ...

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 ...