HD Clinical Trials

Encouraging News from PTC Therapeutics-Pivot-HD Clinical Trial on Drug PTC-518

I’m continuing to share exciting news on Huntington’s disease (HD) drugs that could potentially hold the key to a higher quality of life for Huntington’s Disease (HD)  patients/families.  The next promising drug is PTC-518.

On June 20, 2024, Huntington’s Disease Society of America (HDSA) received an update from PTC Therapeutics about their ongoing trial, PIVOT-HD. PIVOT is testing PTC-518, a small molecule drug, taken as a pill, that lowers huntingtin (HTT) in people with Huntington’s disease (HD).

Dr. Sarah Hernandez, editor-in-chief at HDBuzz.net states, “We know that the genetic cause of HD is an expansion of the genetic code within the HTT gene. There’s an extra bit of genetic message that repeats the letters C, A, and G more times than it should. When someone has 40 or more CAG repeats in their HTT gene, they’ll go on to develop HD, unless researchers can find some way to intervene.”

What does the HTT gene do? The HTT gene provides instructions for making a protein called huntingtin.

We Can Never Lose HOPE….

I’m a Huntington’s disease (HD) advocate, President of the HDSA San Francisco Bay Area Chapter, a blogger and an author.  Visit my website:  https://theresecrutchermarin.com

 

 

 

 

 

 

 

 

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