Huntington's Disease

Huntington's disease (HD) is a progressive, neurodegenerative genetic disorder that affects the brain, leading to motor dysfunction, cognitive decline, and psychiatric disturbances. It is caused by a trinucleotide repeat expansion in the Huntington (HTT) gene, which leads to the production of an abnormal form of the protein huntingtin. This abnormal protein gradually damages neurons, particularly in the striatum and cortex of the brain.

Given the involvement of the striatum and cortex in the pathophysiology of Huntington’s disease scientists often focus their research on rescuing the degeneration of medium spiny neurons – the most affected neurons in the striatum. To this end, Muriphys offers a precise and reproducible intraparenchymal (IPa) drug product delivery into the striatum and thalamus.

Huntington’s disease is characterized by motor, cognitive and psychiatric symptoms.

To compliment our CNS delivery, we offer behavioral tests that encompass all aspects of the disease, including:

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