Synonyms CEREBRAL PALSY, SPASTIC QUADRIPLEGIC, 3, FORMERLY, CPSQ3, FORMERLY, Cerebral Palsy, Spastic Quadriplegic 3, Formerly + [5] |
Introduction An autosomal recessive subtype of hereditary spastic paraplegia caused by mutation(s) in the AP4M1 gene, encoding AP-4 complex subunit mu-1. |
Target |
Mechanism AP4M1 stimulants |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 3 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target |
Mechanism AP4B1 modulators |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date01 Aug 2025 |
Sponsor / Collaborator BlackfinBio Ltd. [+1] |
Start Date01 Mar 2025 |
Sponsor / Collaborator |
Start Date15 Feb 2023 |
Sponsor / Collaborator [+1] |