Clinical Trial Update: A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients [Phase 1, Phase 2]

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Summary

Clinical trial update for SOBI003 in pediatric Sanfilippo Syndrome Type A (MPS IIIA) reporting completion with results posted.

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Diseases / indications

• Mucopolysaccharidosis Type Iiia
• Sanfilippo Syndrome Type

Drugs mentioned

• SOBI003

Snippet

Sponsor: Swedish Orphan Biovitrum | Conditions: Sanfilippo Syndrome Type A (MPS IIIA) | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2022-02-25