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Metazym (rhASA) in late infantile metachromatic leukodystrophy – Phase 2 extension
2007-006345-40Phase 2Completed
- Sponsor
- Shire
- 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), sponsored by Shire. Primary endpoint: Relative change in GMFM after 52 weeks of treatment. Started Jan 2008; status: completed.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)
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