FDA Approves AADC Deficiency Gene Therapy by PTC Therapeutics

added blog Health

2024-11-28 09:12:36 -

PTC Therapeutics has received FDA approval for its innovative gene therapy targeting Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, a rare genetic disorder affecting dopamine synthesis. This therapy, the first of its kind for this condition, promises to improve motor and cognitive functions in affected children, marking a major breakthrough in rare disease treatment and a...

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