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...
ncmc-shanghai-gene-therapy
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...
ncmc-shanghai-gene-therapy