What is it about?

Huntington disease is a rare neurodegenerative, hereditary, progressive disease of the nervous system caused by a CAG repeat mutation in a known gene named HTT. HTT gene is translated into a protein named huntingtin whose dysfunction causes a number of biochemical abnormalities in cells.

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Why is it important?

Huntington disease is a study model for many other neurodegenerative diseases because it is predictable by a genetic test and overlaps clinical manifestations of well known, yet incurable, diseases like Alzheimer and schizophrenia

Perspectives

Pridopidine may represent the first therapeutic resource for Huntington disease with potential of neuroprotection, that means it may modify the disease course.

Dr Ferdinando Squitieri
Irccs casa sollievo sofferenza and Mendel Institute of Human Genetics

Read the Original

This page is a summary of: Profile of pridopidine and its potential in the treatment of Huntington disease: the evidence to date, Drug Design Development and Therapy, October 2015, Dove Medical Press,
DOI: 10.2147/dddt.s65738.
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