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Phase 2 open-label enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII (Sly disease)
jRCT2051190088Phase 2Active, not recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Jan 2019
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of vestronidase alfa in mucopolysaccharidosis type VII, sponsored by Amicus Therapeutics. Primary endpoint: Reduction in urinary glycosaminoglycans at week 24. Started Jan 2019; status: active, not recruiting.
02
Drugs
vestronidase alfa
03
Conditions
mucopolysaccharidosis type VII
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