Clinical Trial Update: A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously [Phase 1, Phase 2]

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Summary

Open-label Phase 1/2 trial of SBC-103 in MPS IIIB patients to evaluate safety, pharmacokinetics, and pharmacodynamics/efficacy; the study was terminated and res…

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Diseases / indications

• Mucopolysaccharidosis Type Iii Sanfilippo

Drugs mentioned

• SBC-103 (recombinant human N-acetylglucosaminidase (NAGLU) enzyme)

Snippet

Sponsor: Alexion Pharmaceuticals, Inc. | Conditions: Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B | Phase 1, Phase 2 | Status: TERMINATED | Results posted: 2018-04-17