Early symptomatic Early juvenile Metachromatic Leukodystrophy (MLD)
Also known as: 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 |
|---|