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Foundation-directed therapeutic development in Huntington's disease.
Celia Dominguez1, Ignacio Munoz-Sanjuan
1CHDI Management, Inc., Advisors to CHDI Foundation, Inc., 6080 Center Drive, Los Angeles, California 90045, United States.
Journal of Medicinal Chemistry
|January 18, 2014
Summary
Huntington's disease (HD), a neurodegenerative disorder caused by a gene mutation, currently lacks disease-modifying treatments. CHDI Foundation is advancing therapeutic development through collaborations and internal programs to combat this devastating condition.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder.
- It stems from CAG repeat expansion in the huntingtin gene (HTT).
- Pathologically, HD involves neuronal loss in cortical, striatal, and hypothalamic areas, leading to chorea, cognitive decline, and psychiatric issues.
Purpose of the Study:
- To outline the therapeutic development efforts of the CHDI Foundation for Huntington's disease.
- To highlight collaborations and internal programs aimed at finding treatments for HD.
Main Methods:
- Review of foundation-directed therapeutic development strategies.
- Description of collaborative and internal research programs in various stages of development.
Main Results:
- CHDI Foundation is actively pursuing multiple therapeutic avenues for HD.
- These efforts encompass both collaborative projects and internal research initiatives.
Conclusions:
- There are currently no effective disease-modifying treatments for Huntington's disease.
- CHDI Foundation is dedicated to accelerating the development of therapies to treat HD.
- The foundation's approach involves strategic collaborations and internal programs to address this unmet medical need.
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