Award
NIH Reporter #1R21TR006299-01A1
Patient-Centric Clinical Outcome Measures for Multiple Sulfatase Deficiency (MSD)
Recipient
Children's Hospital of Philadelphia
Award Amount
$489,500.00
Ceiling
$489,500.00
Awarded
September 03, 2025
Identifier
1R21TR006299-01A1
The National Center for Advancing Translational Sciences funded a two-year project at Children's Hospital of Philadelphia to evaluate patient- and clinician-reported outcome measures for Multiple Sulfatase Deficiency and help prepare for future clinical trials.
Description
Project to develop and validate patient-centric and clinical outcome measures for Multiple Sulfatase Deficiency, supporting disease progression assessment and future clinical trials.