HD Clinical Trials Archives – Therese Crutcher-Marin

UniQure AMT-130 Shows Promise of Slowing Huntington’s Disease Progression

We are living in an age where there are many possibilities for a drug for to combat the symptoms of Huntington’s disease (HD). It is an exciting time for the HD community around the world and my HOPE is at a new level.

Another drug that silences the mutant huntingtin gene, is AMT-130 developed by uniQure. (uniQure’s gene therapy) AMT-130 involves a harmless virus packaged with genetic material that is designed to lower the amount of huntingtin protein in the brain. AMT-130 is delivered via a single surgery into the brain, with the goal of permanently lowering levels of huntingtin in nerve cells.

In July 2024, uniQure announced promising interim data demonstrating slowing of disease progression in our ongoing Ph. I/II clinical trials.

These type of drugs, that control symptoms of HD, will help HD families have a higher quality to their lives.

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

August 28, 2024 by crutcht

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.