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| Ref Type | Journal Article | ||||||||||||
| PMID | (40294355) | ||||||||||||
| Authors | Mossé YP, Polkosnik G, Pogoriler J, Mattei P, States LJ, Maris JM | ||||||||||||
| Title | Anaplastic Lymphoma Kinase Inhibition Therapy for Hereditary Neuroblastoma. | ||||||||||||
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| Molecular Profile | Treatment Approach |
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| Gene Name | Source | Synonyms | Protein Domains | Gene Description | Gene Role |
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| Therapy Name | Drugs | Efficacy Evidence | Clinical Trials |
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| Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
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| Gene | Variant | Impact | Protein Effect | Variant Description | Associated with drug Resistance |
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| ALK R1275Q | neuroblastoma | predicted - sensitive | Alectinib | Case Reports/Case Series | Actionable | In a clinical case study, Alecensa (alectinib) treatment resulted in a complete response in patient with hereditary neuroblastoma harboring germline ALK R1275Q (PMID: 40294355). | 40294355 |
| ALK R1275Q | neuroblastoma | conflicting | Crizotinib | Case Reports/Case Series | Actionable | In a clinical case study, Xalkori (crizotinib) treatment obtained through a Phase I trial resulted in a complete response, with complete and durable remission for 5 years on therapy, in a pediatric patient with hereditary neuroblastoma harboring germline ALK R1275Q (PMID: 40294355). | 40294355 |