Consider taking part in clinical research, where you can join other families in illuminating the path forward. See if the STARLIGHT Study is right for your child.
Have a confirmed diagnosis of Hunter syndrome (MPS II)
Are between 30 and 71 months of age (2.5 years old to younger than 6 years old) and meet certain cognitive/developmental requirements OR are 6 years of age or older with an IQ of 70 or higher
Are able to undergo lumbar punctures (medical procedures to collect samples of cerebrospinal fluid)
Have not received hematopoietic stem cell transplantation (HSCT) with successful engraftment
Have not received gene therapy treatment in a study
Additional criteria may apply.
Qualified participants will receive study-related care and the investigational drug at no cost. Reimbursement for travel and certain study-related expenses will be available.
This condition, also known as mucopolysaccharidosis type II, occurs mostly in males.1
Symptoms vary but can include stiff joints, broadened facial features, delayed growth, cognitive and developmental decline, enlarged spleen and liver, and hearing loss.1
Hunter syndrome occurs in individuals who do not have (or do not have enough of) an enzyme called iduronate sulfatase.2
Signs and symptoms typically begin in children ages 2 to 4 years.1
One in roughly 100,000 to 170,000 males are diagnosed with Hunter syndrome.1
Iduronate sulfatase is needed to break down chains of sugar molecules called glycosaminoglycans.2
Not having this enzyme causes sugar molecules to build up in various body tissues, which causes damage.2
We need clinical research to find a path toward safe and effective treatment options. The STARLIGHT Study aims to bring us a step closer.
