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Safety and Tolerability of VGB-R04 in Patients With Hemophilia B

Gene Therapy Trial VGB-R04 for Hemophilia B Patients in China Trial ID: NCT05152732Study Title: Safety and Tolerability of VGB-R04 in Patients With Haemophilia BSponsor: Institute of Hematology & ...
aav-gene-therapy

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injection in the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) – Phase III ​

Phase III Trial: GC101 Gene Therapy for Type 2 Spinal Muscular Atrophy (SMA) ClinicalTrials.gov Identifier: NCT06971094Sponsor: GeneCradle Inc.Study Title: Safety and Efficacy Evaluation of GC101 Gen...
aav-gene-therapy

Gene Therapy for Phenylketonuria in China

Gene Therapy for Phenylketonuria in China: A Breakthrough Treatment IntroductionPhenylketonuria (PKU) is a rare genetic disorder that affects the body's ability to break down phenylalanine (Phe), an ...
aav-gene-therapy

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