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

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Dr Jeff Carroll on December 11, 2017

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and is safe and well-tolerated.

A new way of thinking about trials to prevent Huntington's disease

A new way of thinking about trials to prevent Huntington's disease

Dr Jeff Carroll on October 27, 2017

A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur.

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.