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Molecular Profile | APC inact mut |
Therapy | Nirogacestat |
Indication/Tumor Type | desmoid tumor |
Response Type | predicted - sensitive |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
APC inact mut | desmoid tumor | predicted - sensitive | Nirogacestat | Case Reports/Case Series | Actionable | In a clinical case study, Ogsiveo (nirogacestat) resulted in stable disease lasting 9 months in a patient with a patient with a desmoid tumor harboring an inactivating APC mutation (PMID: 32762028). | 32762028 |
PubMed Id | Reference Title | Details |
---|---|---|
(32762028) | Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases. | Full reference... |