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First Brain-Delivered Gene Therapy Approved for AADC DeficiencyThe treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
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 ...
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 ...
PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...
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 ...
A Summerville man became the first person in South Carolina to undergo potentially life-changing gene therapy.
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