EU Recommends Approval for PTC Therapeutics’ Gene Therapy Upstaza
Upstaza, a gene therapy developed by PTC Therapeutics for patients with the genetic condition AADC deficiency, has been recommended for EU approval, putting another test of gene therapy's commercial prospects in the union. Upstaza (eladocagene exuparvovec) will become the first disease-modifying medication for AADC deficiency, as well as the first gene therapy directly delivered into the brain. AADC deficiency is a rare fatal disorder that causes significant disability and suffering in children as early as the first months of life, with physical, mental, and behavioral symptoms including seizures.
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