Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Phase 1/Phase 2
1990-06-01
Menkes Disease is a genetic disorder affecting the metabolism of copper. Patient with this
disease are both physically and mentally retarded. Menkes disease is usually first detected
in the first 2-3 months of life. Infant males born with the disease fail to thrive,
experience hypothermia, have delayed development, and experience seizures. These infants also
have characteristic physical features such as changes of their hair and face. Females may
also have changes in hair and skin color, but rarely have significant medical problems.
Appropriate treatment of Menkes Disease requires that the disease be diagnosed early and
treatment started before irreversible brain damage occurs. The aim of treatment is to bypass
the normal route of absorption of copper through the gastrointestinal tract. Copper must then
be delivered to brain cells and be available for use by enzymes.
Copper histidine is a copper replacement that can be injected directly into the body to avoid
absorption through the gastrointestinal tract. However, studies have shown the genetic
abnormalities causing Menkes disease cannot simply be corrected by copper replacement
injections.
The genetic abnormality causing Menkes disease can vary in its severity. Patients with a
genetic abnormality that may still permit some production of the enzymes required to process
copper may receive benefit from early treatment with copper replacement. However, patients
with severe abnormalities of the genes responsible for copper metabolism may receive no
benefit from copper replacement.
The purpose of this study is to continue to evaluate the effects of early copper histidine in
Menkes disease patients and to correlate specific molecular defects with responses to
treatment.
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Last updated: April 28, 2026
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