UX111 for treating mucopolysaccharidosis type IIIA

UX111 is in clinical development for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA). MPS, also known as Sanfilippo syndrome, is a rare genetic condition that causes fatal brain damage and is a type of childhood dementia. MPS III is caused by the lack of an enzyme that normally breaks down and recycles a large, complex sugar molecule called heparan sulphate. This heparan sulphate accumulates and causes damage to the cells of the body, particularly the brain and spinal cord. MPS IIIA is a subtype of MPS III which is caused by a change in a gene. Developmental delay is usually evident in children with MPS IIIA by age 2-5 years. Mental and motor development peak by 3-6 years of age, after which intellectual decline usually occurs. Death can occur from before the age of 10 or not until the third of fourth decades of life, with the average being around 15 to 20 years of age. There is currently no approved treatment for MPS IIIA. Therefore, there is a high unmet need for treatment options for MPS IIIA.