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

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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.

Important advances in next generation genome editing tools for Huntington's Disease

Important advances in next generation genome editing tools for Huntington's Disease

Mr. Shawn Minnig on October 31, 2016

Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the HD mutation on cells. What's new in this exciting area of research?

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?

EuroBuzz 2016: The Euro-HD Network Meeting in The Hague

EuroBuzz 2016: The Euro-HD Network Meeting in The Hague

Dr Jeff Carroll on September 23, 2016

Here's our roundup of all the science presented at the 2016 European Huntington's Disease Network biennial meeting - one of the biggest meetings of Huntingtons Disease families, scientists and care professionals.

Ultra-rare mutations highlight the importance of the HD gene in brain development

Ultra-rare mutations highlight the importance of the HD gene in brain development

Megan Krench on August 29, 2016

A relatively new technology called exome sequencing has identified a few families with novel mutations in their HD genes. These are different than the mutation that causes HD, but allow researchers to better understand the normal role of the HD gene.

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.

A support system gone wrong - glial cells contribute to HD symptoms

A support system gone wrong - glial cells contribute to HD symptoms

Dr Jackie Johnson on August 02, 2016

A new piece of research has implicated a type of brain cells called glia in the development of HD symptoms. Regular mice injected with glial cells carrying the mutant huntingtin gene in the study developed symptoms associated with HD. Interestingly, this influence appears to work both ways — a mild reduction in disease symptoms was seen when HD mice were treated with normal glial cells.

Slightly long CAG repeats are more common than we thought

Slightly long CAG repeats are more common than we thought

Dr Jeff Carroll on July 05, 2016

Everyone with Huntington's disease has inherited the same type of mutation from their mother or father, an extra-long repetitive stretch of the sequence C-A-G in their HD gene. But the length of the mutation varies between individuals, and longer repeats are associated with earlier onset of symptoms. A huge new international study reveals that slightly longer-than-normal CAG stretches are much more common than we thought. Surprisingly, this turns out to be good news.

Early exposure to the HD protein may cause life-long symptoms

Early exposure to the HD protein may cause life-long symptoms

Mr. Shawn Minnig on June 28, 2016

When the ‘healthy’ HD gene functions as it should, one of its many jobs is in the development of normal embryos. Researchers have long assumed that the ‘mutant’ HD gene inherited by people with HD is still able to do this job, since HD patients develop normally and don’t show signs until later in life. A surprising new finding suggests we may have to think carefully about this assumption!