TRIALSITEIQ PUBLIC FEASIBILITY SCORECARD · GRADE C

NCT02716246: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Sponsor: Ultragenyx Pharmaceutical Inc · Phase: PHASE2/PHASE3 · Area: CARDIOLOGY_METABOLIC · Target Enrollment: 36 pts

Competing Trials Overlap

3 Active Trials

Excess Burn Exposure

$9,882,976

Rescue Site Savings

+$6,521,144

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