Mini brains grown in a dish shed light on Huntington’s disease and how we might treat it
Stem cells grown in 3D in a research lab can mimic some features of Huntington’s disease (HD). They also hold promise for transplantation studies to potentially add back cells that are lost in HD. But what would happen to those new cells? Would they get along with the cells still in the brain that have […]
Steady progress from uniQure – promising data to end the year
With the holidays approaching, welcome news arrived on December 19th in a press release from uniQure. The latest data from the HD-GeneTRX studies of AMT-130, an experimental huntingtin-lowering gene therapy, shows that the drug still appears to be safe over the course of a few years. Since the number of participants is very small, we […]
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 […]
Hereditary Disease Foundation (HDF) conference 2024 – Day 4
We’re back for the last day of the Hereditary Disease Foundation conference! Serendipitous finding? Up first is HDBuzz co-founder and editor emeritus, Jeff Carroll. Jeff’s lab studies HD in mice and cells in a dish and investigates different potential treatments. The first story Jeff is telling us about is developing tools that lower HTT. He’s […]
Hereditary Disease Foundation (HDF) conference 2024 – Day 3
We’re back for Day 3 of the Hereditary Disease Foundation (HDF) conference! First up is a session on RNA dynamics – what’s that?! Read on to find out! Different HTT forms have different effects Up first is Gill Bates, who will tell us about her work in understanding how somatic expansion causes disease and investigating […]
Hereditary Disease Foundation (HDF) conference 2024 – Day 2
Welcome to Day 2 of the Hereditary Disease Foundation (HDF) conference! The morning was spent listening to an interview between a neurologist and their patient living with HD. All HDF meetings begin this way, to better connect scientists with the people who matter most, those living with HD. Different flavors of HTT Up first is […]
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