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Intracerebral AAV-NAGLU gene therapy for Sanfilippo type B syndrome
ISRCTN19853672Phase 2Completed
- Sponsor
- Institut Pasteur
- Start
- Sep 2013
- Modality
- RNA-Targeting Small Molecules → Nucleic Acid-Directed Small Molecules → Small Molecules, Small Molecules, Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ABO-101, tacrolimus and mycophenolate mofetil in mucopolysaccharidosis type IIIB, sponsored by Institut Pasteur. Started Sep 2013; status: completed.
02
Drugs
ABO-101tacrolimusmycophenolate mofetil
03
Conditions
mucopolysaccharidosis type IIIB
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