Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Status:
Completed
Trial end date:
2017-10-16
Target enrollment:
Participant gender:
Summary
Study to evaluate the safety and tolerability of intravenous (IV) administration of SBC-103
in participants with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) with
evaluable signs or symptoms of developmental delay.