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Phase I/II study of intracisternal RGX-111 in severe mucopolysaccharidosis type I
RBR-758cbnPhase 2Not yet recruiting
- Sponsor
- Hospital de Clínicas de Porto Alegre
- Start
- Nov 2018
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of RGX-111 in mucopolysaccharidosis type I and Mucopolysaccharidosis Type I Hurler (2 cohorts), sponsored by Hospital de Clínicas de Porto Alegre. Planned start Nov 2018; status: not yet recruiting.
02
Drugs
RGX-111
03
Conditions
mucopolysaccharidosis type IMucopolysaccharidosis Type I Hurler
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