Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
The stormy trial updates that hung over the Huntington’s disease (HD) field in 2021 have certainly parted, making way for the bright and clear forecast we’ve had so far in 2024! Close on the heels of recent positive trial news from Sage Therapeutics, PTC Therapeutics, Wave Life Sciences, and uniQure, we’ve received more encouraging results […]
Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial
New data from uniQure, who developed a one-and-done gene therapy for Huntington’s disease (HD) called AMT-130, indicates that the drug is relatively safe and might be able to slow down signs and symptoms of HD. AMT-130 is currently under investigation in Phase I/II clinical trials in Europe and the US which are mainly focused on […]
Positive news from Wave Life Sciences SELECT-HD trial
On 25th June 2024, Wave Life Sciences shared the results of their SELECT-HD clinical trial. This trial tested a therapy called WVE-003, designed to only lower the expanded, harmful form of the huntingtin (HTT) protein found in people with Huntington’s disease (HD). The data released today give positive updates on the safety of this therapy […]
No pivot needed for PTC-518
On June 20, 2024, we received an update from PTC Therapeutics about their ongoing trial, PIVOT-HD. PIVOT is testing PTC-518, a small molecule drug, taken as a pill, that lowers huntingtin (HTT) in people with Huntington’s disease (HD). Their most recent update shared data from people who had been on the drug for 12 months. […]
SURVEYOR opens the door for drugs that treat cognition
Sage Therapeutics released a press statement on June 11th that focuses on the main results of a study called SURVEYOR, aimed at studying cognition (thinking) in Huntinton’s disease (HD) and testing the safety of a drug called dalzanemdor (previously SAGE-718). Let’s talk about what we know and what’s next! Amplifying nerve cell messages to improve […]
The VIP section: velvet ropes for the brain and how to get in
Two separate research groups recently published work on the blood-brain barrier (BBB). You can think of the BBB like a bouncer that keeps the riffraff out of the VIP section that is your brain. One group advanced how the brain’s barrier is modeled in the lab using stem cells. Another group developed a harmless virus […]
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