A clinical trial for Sanfilippo Syndrome Type A is currently enrolling patients.
The Lyrics Study, HGT-SAN-093, is evaluating the safety and efficacy of intrathecal delivery of an enzyme replacement therapy on cognitive function in pediatric patients with Early Stage MPS IIIA Disease (Sanfilippo Syndrome Type A.) There are currently no effective disease therapies currently approved for this devastating and disabling disease.
Each patient must meet the following criteria to be enrolled in this study.
1. Documented MPS IIIA diagnosis.
2. Between 12 and 48 months of age at the time of enrollment.
3. The patient has a DQ score equal to less than 60%.
IF you have interested in the study and would like to learn more you can contact Dr. David Alexanderian directly at Dalexanderian@shire.com.