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

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

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

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

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

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

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

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 a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic ...

Bayer has become the latest in a long line of drug developers to give up on Huntington’s disease after the German pharma ...

Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...

Modifying Treatment for AADC Deficiency in the United States SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024 / ...