Gene Therapy in patients with Mucopolysaccharidosis disease
A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects withMucopolysaccharidosis type VI (MPS VI) Using Adeno-Associated ViralVector 8 to Deliver the human ARSB gene to Liver. A Phase I/II Open Label, Dose Escalation, Safety Study in Subjects withMucopolysaccharidosis type VI ...
The clinical trial will be conducted on patients withMucopolysaccharidosis Type VI. MPS VI is characterized by growthretardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] The clinical trial will be conducted on patients withMucopolysaccharidosis Type VI. MPS VI is charac ...