Articles with the topic: featured

No pivot needed for PTC-518

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.

Dr Sarah HernandezJune 21, 2024

SURVEYOR opens the door for drugs that treat cognition

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.

Dr Leora FoxJune 12, 2024

The VIP section: velvet ropes for the brain and how to get in

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.

Dr Sarah HernandezJune 10, 2024

Two birds, one stone: HTT-lowering drugs also target CAG expansions

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.

Dr Sarah HernandezMay 21, 2024

Hats off to brain donors on Brain Donation Awareness Day

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.

Dr Sarah HernandezMay 07, 2024

A sprinkling of good news for the treatment of HD chorea

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.

Dr Leora FoxMay 03, 2024

A new era for HDBuzz

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.

Dr Sarah Hernandez, Dr Leora Fox, and Dr Rachel HardingMay 01, 2024

How many is too many? Exploring the toxic CAG threshold in the Huntington’s disease brain

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.

Dr Chris KayApril 21, 2024

Cry your eyes out: detecting huntingtin in tears

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.

Dr Sarah HernandezApril 10, 2024