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EU CHMP Recommends Approval of PTC Therapeutics Gene Therapy for Treatment of AADC Deficiency

EU CHMP Recommends Approval of PTC Therapeutics Gene Therapy for Treatment of AADC Deficiency
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National taiwan university hospitalT ai peiStuart peltzLiang hwuEuropean unionEuropean commissionMedicinal productsHuman useEuropean medicines agencyPaul wuh liang hwuNational taiwan university

PTC Therapeutics Receives Positive CHMP Opinion for Upstaza™ for the Treatment of AADC Deficiency

/PRNewswire/ PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the.

National taiwan university hospitalT ai peiUnited statesJeanine clementeKylie okeefeStuartw peltzLiang hwuEuropean commissionAdministration of upstazaLancet child adolesc healthChild neurology societyTherapeutics incEuropean unionEuropean medicines agencyCommittee for medicinal products human useMedicinal products

Novel gene therapy improves outcomes in children with rare neurometabolic disorder

Novel gene therapy improves outcomes in children with rare neurometabolic disorder
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Stuartw peltzLiang hwuTherapeutics incNational taiwan university hospitalPaul wuh liang hwu

Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy

Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy
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United statesKylie okeefeLiang hwuJane bajDrug administrationLancet child adolesc healthTherapeutics incNational taiwan university hospitalPaul wuh liang hwuNational taiwan universityEuropean medicines agencyMedicinal productsHuman useBiologics license applicationMore informationPrivate securities litigation reform act

Targeting the Putamen with Gene Therapy Leads to Sustained Improvements in Motor and Non-Motor Functions in Children with AADC Deficiency

Targeting the Putamen with Gene Therapy Leads to Sustained Improvements in Motor and Non-Motor Functions in Children with AADC Deficiency
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National taiwan university hospitalT ai peiUnited statesKylie okeefeStuartw peltzLiang hwuJane bajEuropean molecular biology organization journalTherapeutics incEuropean molecular biology organizationChief executive officerPaul wuh liang hwuNational taiwan universityEuropean medicines agencyMedicinal productsHuman use