Publication | Closed Access

Final results of the phase 1/2, open-label clinical study of intravenous recombinant human N-acetyl-α-d-glucosaminidase (SBC-103) in children with mucopolysaccharidosis IIIB

Phase 1/2Translational MedicineMucopolysaccharidosis IiibAutoimmune DiseaseMedicineImmunodeficienciesPathologyFinal ResultsLysosomal Storage Disease