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Health

Existing USFDA-approved drug shows promise for 2 rare genetic disorders

November 14, 2024 09:38 AM

New Delhi, Nov 14 || A team of Canadian researchers has found an existing US Food and Drug Administration-approved drug that could significantly improve the quality of life for patients affected with Sandhoff and Tay-Sachs diseases -- two rare genetic disorders.

Sandhoff and Tay-Sachs diseases cause progressive damage to nerve cells in the brain and spinal cord.

There is currently no cure for both disorders.

After years of investigating the diseases’ underlying mechanisms, the research at McMaster University identified a potential therapeutic compound: 4-phenylbutyric acid (4-PBA).

4-PBA is an FDA-approved drug that was initially developed for another condition.

Suleiman Igdoura, a professor of biology and pathology at the varsity stated that Sandhoff and Tay-Sachs “are devastating diseases that are marked by progressive loss of motor functions -- from sitting, standing, and swallowing to even breathing -- as neurons in the nervous system die”.

In the study, published in the journal Human Molecular Genetics, the team tested 4-PBA in a mouse model of the disease. The results showed that 4-PBA significantly improved motor function, extended lifespan, and increased the number of healthy motor neurons.

Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often proving fatal within a few years.

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