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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…
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)…
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…
Two birds, one stone: HTT-lowering drugs also target CAG expansions
Huntingtin (HTT)-lowering and somatic expansion have been two of the hottest topics in Huntington’s disease (HD) research in the past decade. Recent work from a…
Highlights from the 2024 CHDI HD Therapeutics Conference
Highlights from the 2024 CHDI HD Therapeutics Conference || Palm Springs 26 – 29 February 2024 Including Clinical Trial updates from PTC Therapeutics, Wave Life…