Abstract
Menkes disease (MD) is a X-linked recessive disorder caused by variants in the copper transporter gene, ATP7A. Although MD patients appear asymptomatic at birth, they develop significant neurological signs and symptoms such as seizures, hypotonia, and failure to thrive approximately 6-10 weeks after birth. Death by 3 years of age is the typical outcome in untreated MD. Copper histidinate (CUTX-101) is being investigated for treatment of this illness.
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
