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Full Steam Ahead: uniQure’s On Track With Hope for Accelerated Approval of Huntington’s Disease Drug
Latest News
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No pivot needed for PTC-518
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
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SURVEYOR opens the door for drugs that treat cognition
Sage Therapeutics announced results from SURVEYOR, a trial that looked at cognitive changes in people with HD, and tested short-term safety of dalzanemdor. The study was small but met key goals, and additional trials are in progress.
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The VIP section: velvet ropes for the brain and how to get in
New work from two groups advances tools to model the blood-brain barrier and could improve the way drugs are delivered for brain diseases. This builds on our ability to study, and eventually treat, Huntington’s disease.
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Two birds, one stone: HTT-lowering drugs also target CAG expansions
In a surprising twist, oral HTT-lowering drugs also slow somatic expansion in the HTT gene. A new study that used cells in a dish for this fortuitous discovery identified the gene PMS1 as a key player in the slowing of CAG expansions.
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Hats off to brain donors on Brain Donation Awareness Day
May 7 is Brain Donation Awareness Day. Today we highlight the selfless donation that many HD families have made, sending our gratitude, sharing research updates made with those precious brains, and detailing resources for brain donation.
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A sprinkling of good news for the treatment of HD chorea
A new form of the chorea drug valbenazine (INGREZZA) has been approved by the United States Food and Drug Administration (FDA) for those who have difficulty swallowing pills.
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A new era for HDBuzz
For over a decade HDBuzz has reported unbiased news about Huntington’s disease research and trials. As our beloved Ed and Jeff step back, Rachel, Sarah, and Leora look forward to upholding the HDBuzz mission.
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How many is too many? Exploring the toxic CAG threshold in the Huntington’s disease brain
New work from researchers in London uses mice to narrow in on the number of CAG repeats needed to cause symptoms of Huntington’s disease. Their work points to fewer than 185 CAGs as a threshold.
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Cry your eyes out: detecting huntingtin in tears
Is someone cutting onions? Expanded huntingtin can now be detected in tears to help scientists track disease progression.
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The director’s cut: how CAG repeats change the editing of genetic messages
Scientists in Massachusetts have recently advanced our understanding of how repetitive sequences in DNA can disrupt the creation and editing of genetic messenger molecules in cells, and how this could lead to the production of harmful proteins.