Copper Histidinate Treatment for Menkes Disease

Summary

This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.

Timeline

Start

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Primary completion

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Completion

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Outcome

Met primary endpoint

release “In both pre-specified primary and secondary efficacy analyses, treatment with CUTX-101 demonstrated a significantly greater median overall survival (OS) compared to untreated historical control patients.” sentynl.com ↗

Drugs

Indications