Pre-symptomatic Early juvenile Metachromatic Leukodystrophy (MLD)
Drug | Drug Name | Drug Description |
---|---|---|
DB17538 | Atidarsagene autotemcel | A gene therapy comprising autologous CD34+ enriched stem cells encoding the human arylsulfatase A (ARSA) gene used to treat metachromatic leukodystrophy (MLD). |
Drug | Drug Name | Target | Type |
---|---|---|---|
DB17538 | Atidarsagene autotemcel | Human Arylsulfatase A mRNA | target |
Drug | Drug Name | Phase | Status | Count |
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