Alström Syndrome (AS) Alström Syndrome (AS) is an ultra-rare genetic disorder characterized by a spectrum of progressive symptoms, including vision and hearing loss, obesity, type 2 diabetes, cardio...
Bardet-Biedl Syndrome (BBS) Bardet-Biedl Syndrome (BBS) is a rare, multisystem genetic disorder characterized by symptoms such as retinal degeneration, obesity, polydactyly, kidney abnormalities, and...
Gene Therapy Clinical Trial for Hemophilia B: ZS801 in China Trial ID: NCT05641610Study Title: A Non-Randomized, Open-Label, Dose-Escalation, Phase I/II Study to Evaluate the Safety, Tolerability, Kin...
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...