Last update 21 Nov 2024
H-ABC Syndrome (Myrtelle)
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms H-ABC (Myrtelle), Hypomyelination With Atrophy of the Basal Ganglia and Cerebellum (Myrtelle) |
Target |
Mechanism TUBB4A inhibitors(tubulin beta 4A class IVa inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization  Myrtelle, Inc.Startup |
Active Organization Myrtelle, Inc.Startup |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Leukodystrophy, Hypomyelinating, 6 | Preclinical | US | Myrtelle, Inc.Startup | 25 Apr 2024 |
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Clinical Result
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