Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The 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.
PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...
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., ...
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 ...
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