Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.

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Articles with the topic: clinical-trial

Update confirms Huntington's disease 'gene silencing' trial on track

Update confirms Huntington's disease 'gene silencing' trial on track

Dr Jeff Carroll on October 15, 2017

Ionis Pharmaceuticals launched the first ever trial of a huntingtin-lowering drug – sometimes called a 'gene silencing drug' – in late 2015. In a significant update, the company has announced two important milestones: the trial is now fully recruited, and an 'open-label extension' will be activated for the volunteers in the current trial. While nothing is guaranteed, this bodes well for the future of this important program.

Precision huntingtin-lowering drug trials target the mutant protein

Precision huntingtin-lowering drug trials target the mutant protein

Dr Michael Flower on August 25, 2017

A new exciting chapter in Huntington’s disease (HD) treatment is just beginning – WAVE Life Sciences have announced PRECISION-HD1 and 2, clinical trials of two new drugs that lower the mutant Huntington’s disease protein. We’re excited about this novel approach to huntingtin lowering therapies, but these are early days and we’ve got a long way to go to show they’re safe and effective in people.

New study reveals a potential HD biomarker

New study reveals a potential HD biomarker

Leora Fox on June 07, 2017

What if a blood test could provide information about the status and course of HD? This is the premise of seeking HD biomarkers; they may someday help guide treatment decisions and predict how symptoms will change. A team of researchers spanning multiple countries recently analyzed blood, brain images, and clinical exams from the TRACK-HD study. They found that blood levels of a protein called neurofilament light chain corresponded with the severity of HD, making it a potential biomarker.

FDA approves a new drug for symptoms of Huntington's disease

FDA approves a new drug for symptoms of Huntington's disease

Dr Jeff Carroll on April 05, 2017

Significant news for the Huntington's disease community this week, as the USA's drug regulator, the FDA, has formally approved Austedo, also known as deutetrabenazine, for prescribing in HD. This modified form of tetrabenazine helps control chorea, the jerky movements often found in HD patients, but is taken twice rather than three times a day.

Pfizer Amaryllis trial ends in disappointment: no improvement in Huntington's disease symptoms

Pfizer Amaryllis trial ends in disappointment: no improvement in Huntington's disease symptoms

Dr Ed Wild on December 16, 2016

Pfizer has announced that the first-pass analysis of its 'Amaryllis' trial, testing a PDE-10 inhibitor drug, shows the drug did not meet its target of improving Huntington's disease symptoms. As a result, the open-label extension study will be stopped. This is not the news we'd been hoping for, but we've learned a lot about HD along the way.

Sorry folks, the PRIDE-HD trial did NOT show that Pridopidine slows the progression of Huntington's disease

Sorry folks, the PRIDE-HD trial did NOT show that Pridopidine slows the progression of Huntington's disease

Dr Jeff Carroll on September 30, 2016

A recent press release from Teva Pharmaceuticals has the HD community excited, claiming "Pridopidine Demonstrates Slowing of Progression of Huntington Disease in PRIDE-HD Study". What's pridopidine, and what can we really say about HD progression in patients treated with it?

Deutetrabenazine for Huntington's disease: a positive trial but the FDA says no? Not exactly...

Deutetrabenazine for Huntington's disease: a positive trial but the FDA says no? Not exactly...

Dr Peter McColgan on August 17, 2016

A scientific paper declares positive results for a trial of deutetrabenazine in Huntington's disease, but the headlines report the FDA has rejected the drug. Confusing stuff! The reality is positive overall for this new way of treating uncontrollable movements in HD, but patience will be needed to see where it all ends up.

Planting trees together: The 2016 Huntington's Disease Society of America Convention

Planting trees together: The 2016 Huntington's Disease Society of America Convention

Dr Jeff Carroll on June 13, 2016

Nearly a thousand HD family members converged on Baltimore, Maryland for the 2016 Huntington’s Disease Society of America’s Annual Convention. We normally don’t write reports from patient and family conferences, but there was something special about the atmosphere of this year’s Convention that compelled us to pen a brief update.

Thinking clearly about the earliest symptoms of HD and which brain regions control them

Thinking clearly about the earliest symptoms of HD and which brain regions control them

Dr Tamara Maiuri on April 18, 2016

Cognitive deficits, or difficulties thinking clearly, often appear well before the traditional clinical diagnosis of Huntington’s disease (HD). While many contend that the earliest cognitive deficits are caused by damage to the striatum – a structure deep in the brain known to be severely affected in HD – recent evidence suggests that this claim may paint an incomplete picture of the widespread changes occurring in the brains of HD patients during the very early stages of the disease.