Clinical Trial Update: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease [Phase 2]

View source ↗

Summary

Phase 2 randomized trial assessing safety and efficacy of rhHNS administered via IDDD in pediatric Sanfilippo syndrome (MPS IIIA); study completed with posted r…

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Sanfilippo Syndrome

Drugs mentioned

• rhHNS (recombinant human iduronidase? (Note: rhHNS refers to a recombinant human heparan/N-sulfoglucosaminidase surrogate; if specific generic name is not provided, set as null))

Snippet

Sponsor: Shire | Conditions: Sanfilippo Syndrome | Phase 2 | Status: COMPLETED | Results posted: 2017-03-03