Mucopolysaccharidosis Type II Natural History

Launched by REGENXBIO INC. · May 7, 2018

Keywords

ClinConnect Summary

No description provided

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Documented diagnosis of MPS II confirmed by enzyme activity as measured in plasma, fibroblasts, or leukocytes
• • 2. The subject has at least one of the neurocognitive assessments listed below, which occurred prior to age 6 and in or after 2006 in their medical records.
• • 1. Bayley Scales of Infant and Toddler Development (BSID), any version
• • 2. Differential Ability Scale (DAS), any version
• • 3. Griffiths Mental Development Scale (GMDS), any version
• • 4. Kaufman Assessment Battery for Children (KABC), any version
• • 5. Kinder Infant Development Scale (KIDS)
• • 6. Kyoto Scale of Psychological Development (KSPD), any version
• • 7. Leiter International Performance Scale (LIPS), any version
• • 8. Mullen Scales of Early Learning (MSEL), any version
• • 9. Vineland Adaptive Behavior Scales (VABS), any version
• • 10. Wechsler Intelligence Scale for Children (WISC), any version
• • 11. Wechsler Preschool and Primary Scale of Intelligence (WPPSI), any version
• • 3. If the subject has undergone hematopoietic stem cell transplantation (HSCT), they must have at least one neurocognitive assessment prior to HSCT.