Menkes Disease: Clinical Trials

The NICHD conducts and supports a variety of clinical research studies related to Menkes disease. Select a link below to learn more about these projects.

Featured NICHD Clinical Trials on Menkes Disease

  • Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency. This study is investigating the effectiveness, side effects, and dosage of copper histidine treatment for patients with copper deficiency. It is also collecting patients' medical histories to allow researchers to study possible genetic and nongenetic origins of copper deficiency.
  • Copper Histidine Therapy for Menkes Diseases. Early, presymptomatic therapy with copper injections has been associated with improved overall survival and, in some patients, with vastly better neurological outcomes in comparison to the usual natural history of this disorder. These patients typically have a distinctive genetic makeup. This study continues to evaluate the effects of early copper histidine in Menkes disease patients and to correlate specific molecular defects with responses to treatment.

NICHD Clinical Trials Search Results

Information on current NIH-sponsored clinical trials on Menkes disease is available at the link below or by calling 800-411-1222.

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