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AMT-110 Gene Therapy for Sanfilippo Type B
NCT03300453Phase 2Completed
- Sponsor
- uniQure
- Start
- Sep 2013
- Primary completion
- Nov 2019
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
01
Overview
A Phase 2 trial of rAAV2/5-hNAGLU in Sanfilippo syndrome type B, sponsored by uniQure. Started Sep 2013; status: completed.
02
Drugs
rAAV2/5-hNAGLU
03
Conditions
Sanfilippo syndrome type B
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