Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory oversight plays in clinical trials and the approval of medicines for Huntington’s disease.
Although pridopidine has suffered four negative trials for HD, the message from Prilenia continues to be positive. What is hope and what is hype in this sixteen-year quest for regulatory approval?
More good news for the HD community from uniQure, whose one-and-done gene therapy appears safe and shows hints of slowing down signs and symptoms of the disease
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
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.
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
