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N-Acetyl-L-Leucine in GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
DRKS00017539Phase 2Active, not recruiting
- Sponsor
- IntraBio
- Start
- Jul 2019
- Modality
- Recombinant Proteins and Enzymes, Peptides
- Countries
- 4
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Overview
Phase 2, single-blind trial of trenonacog alfa in GM2 gangliosidosis, Tay-Sachs disease (TSD) and Sandhoff disease (2 cohorts), sponsored by IntraBio. Started Jul 2019; status: active, not recruiting.
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Drugs
trenonacog alfa
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Conditions
GM2 gangliosidosisTay-Sachs disease (TSD)Sandhoff disease
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