MPS I Clinical Trials in Pittsburgh, Pennsylvania
2 recruitingPittsburgh, Pennsylvania
Showing 1–2 of 2 trials
Recruiting
Phase 2Phase 3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Mucopolysaccharidosis IIIMPS IIIASanfilippo Syndrome+1 more
Ultragenyx Pharmaceutical Inc36 enrolled5 locationsNCT02716246
Recruiting
Longitudinal Study of Neurodegenerative Disorders
Gaucher DiseaseBatten DiseaseLeukodystrophy+24 more
University of Pittsburgh1,500 enrolled1 locationNCT03333200