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...
Clinical Trial Update: SKG0201 Gene Therapy for Spinal Muscular Atrophy Type 1 (SMA 1) Trial ID: NCT06191354Study Title: A Multicenter, Open, Dose-Escalation Clinical Study Evaluating the Safety, Init...
IntroductionChimerix, a prominent biopharmaceutical firm, has declared that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for Dordaviprone (ONC201). The medic...
Gene Therapy for Gaucher’s Disease in ChinaGaucher's disease is a rare inherited condition that happens when the body doesn't have enough of the glucocerebrosidase (GBA) enzyme, leading to harmful f...
Gene Therapy for Wilson's Disease in China Introduction Wilson’s disease is a rare genetic disorder that causes excessive copper accumulation in the liver, brain, and other vital organs. With...
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 ...
Best Cancer Hospitals in China China has become a world leader in medical innovation, especially in oncology. The nation has, over the past decade, made tremendous progress in biotechnology, fo...
Cancer remains one of the most challenging diseases to treat, requiring innovative and effective therapies. One of the most promising advancements in cancer treatment is Chimeric Antigen Receptor (CA...
On November 8, 2024, the Food and Drug Administration sanctioned obecabtagene autoleucel (Aucatzyl, Autolus Inc.), a CD19-targeted genetically engineered autologous CAR T cell therapy, for people with...