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Metazym (rhASA) multiple-dose study in late infantile MLD with high residual voluntary function
2007-007165-20Phase 2Completed
- Sponsor
- Takeda
- Start
- Jan 2008
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of cebsulfase alfa in metachromatic leukodystrophy (MLD) and leukodystrophies, sponsored by Takeda. Primary endpoint: Relative change in GMFM after 26 weeks of treatment. Started Jan 2008; status: completed.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)leukodystrophies
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