Synonyms CAPILLARY MALFORMATION-ARTERIOVENOUS MALFORMATION, CAPILLARY MALFORMATION-ARTERIOVENOUS MALFORMATION (disorder), CM-AVM + [10] |
Introduction An autosomal dominant inherited vascular disorder associated with loss of function mutations in the RASA1 or EPHB4 gene, encoding Ras GTPase-activating protein 1 or ephrin type-B receptor 4, respectively. It is characterized by cutaneous capillary malformations, often in association with arteriovenous malformations and arteriovenous fistulas, which may lead to abnormal bleeding, migraine headaches, seizures, and heart failure. |
Target |
Mechanism SOS1 inhibitors |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhaseDiscovery |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date25 Apr 2022 |
Sponsor / Collaborator |
Start Date01 Jan 2020 |
Sponsor / Collaborator- |
Start Date30 Apr 2018 |
Sponsor / Collaborator- |