Prilenia Phase 3 Trial with Pridopidine – Why is it Different?

– September 13th, 2022

For all those who have been following Huntington’s disease research for some time, Pridopidine is an “old acquaintance”.

To date, several studies have been conducted with this drug, looking closely to its effect on dopamine. Although they have helped us to confirm the safety and good tolerability of pridopidine, none of these trials has been completely successful.

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UniQure: the Highs and Lows of AMT-130

– September 9th, 2022

UniQure is a company with an expertise in gene therapy that has long been involved in the search for HD treatment options. Their preclinical studies in animal models showed that AMT-130, the gene therapy product candidate for HD, reduced the mutant huntingtin protein and was associated with a decrease in the progression of Huntington’s disease signs.

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PIVOT-HD, PTC Therapeutics Phase 2 Trial to Lower Huntingtin

– September 8th, 2022

PTC Therapeutics is a US biopharmaceutical company specialized in the development of  gene therapies for rare diseases. The team at PTC Therapeutics has screened more than 300,000 molecules in the search for an effective therapeutical approach to HD, one  that can lower huntingtin, can be administered orally and can have an impact inside and outside of the brain. 

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Suspending is not Ending: Updates about the Novartis VIBRANT-HD trial

– September 8th, 2022

At the end of August, Novartis has written a letter to the HD community to explain the reasons behind their decision to temporarily suspend dosing of Branaplam in the Phase 2b VIBRANT-HD study with adults with HD.

While we know that this kind of news always comes as a shock to all families living with HD, we must remember that we are protecting and safeguarding the health of the brave and generous patients who have voluntarily signed up for this research.

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Another Vibrant Possibility for Huntington’s Disease Treatment

– March 17th, 2022

Novartis Pharmaceuticals is recruiting participants for their phase IIb clinical trial called VIBRANT-HD. This study will investigate the huntingtin lowering drug Branaplam, which was originally developed for Spinal Muscular Atrophy, a devastating paediatric genetic disease.

Novartis will test Branaplam in patients with early manifest HD to check the safety of the drug in HD and learn what is the right dose to lower huntingtin, the harmful protein.

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Updates about Roche GENERATION-HD1 and Tominersen: it’s not over until it’s over

– February 3rd, 2022

The pharmaceutical company Roche has recently shared with the HD community further details about the phase III clinical trial GENERATION HD-1. This clinical trial involved nearly 800 HD patients from around the world and stopped dosing in March 2021 following the advice from an Independent Data Monitoring Committee. 

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