Latest news

uniQure gets the green light to resume testing HD gene therapy

uniQure gets the green light to resume testing HD gene therapy

Following a 3-month pause in enrollment due to concerns about side effects, uniQure shared the good news that their trial of the HD gene therapy AMT-130 will continue as planned, with new safety measures in place.

Dr Leora FoxNovember 03, 2022

Update on the PTC Therapeutics PIVOT-HD Trial

Update on the PTC Therapeutics PIVOT-HD Trial

Last week, PTC Therapeutics released a statement sharing that recruitment of participants into the US arm of the PIVOT-HD trial has been paused. In this article, we will lay out exactly what is known and what this announcement means.

Dr Rachel Harding and Dr Leora FoxNovember 03, 2022

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Forward momentum for Roche and Wave in latest news about huntingtin-lowering trials

Two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. Let’s talk about the history and the caveats surrounding these hopeful pieces of news.

Dr Jeff Carroll and Dr Leora FoxSeptember 30, 2022

Focusing in on fibrils; scientists give us a glimpse of huntingtin protein clumps

Focusing in on fibrils; scientists give us a glimpse of huntingtin protein clumps

Scientists have used powerful microscopes to look at sticky fragments of the Huntington’s disease protein, shedding light on these structures which are thought to drive the disease

Dr Rachel HardingSeptember 08, 2022

Hereditary Disease Foundation (HDF) conference 2022 – Day 4

Hereditary Disease Foundation (HDF) conference 2022 – Day 4

Read updates from clinical trials and scientific research on Huntington’s disease from Day 4 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022

Dr Sarah Hernandez and Dr Rachel HardingSeptember 02, 2022

Hereditary Disease Foundation (HDF) conference 2022 – Day 3

Hereditary Disease Foundation (HDF) conference 2022 – Day 3

Read updates from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022

Dr Rachel Harding, Dr Leora Fox, Dr Sarah Hernandez, and Dr Jeff CarrollSeptember 01, 2022

Hereditary Disease Foundation (HDF) conference 2022 – Day 2

Hereditary Disease Foundation (HDF) conference 2022 – Day 2

Read updates from clinical trials and scientific research on Huntington’s disease from Day 2 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022

Dr Leora Fox, Dr Rachel Harding, and Dr Sarah HernandezAugust 31, 2022

Hereditary Disease Foundation (HDF) conference 2022 – Day 1

Hereditary Disease Foundation (HDF) conference 2022 – Day 1

Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022

Dr Rachel Harding and Dr Sarah HernandezAugust 31, 2022

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials

After receiving a high dose of uniQure’s gene therapy for Huntington’s disease, a few patients experienced serious side effects, but are now recovering. HDBuzz explores what this means for the experimental huntingtin-lowering drug, AMT-130.

Dr Sarah Hernandez and Dr Rachel HardingAugust 29, 2022

Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam

Sad news from Novartis: dosing suspended in VIBRANT-HD trial of branaplam

HDBuzz is saddened to share the news that the VIBRANT-HD trial of the oral huntingtin-lowering drug, branaplam, has been temporarily suspended for the safety of participants.

Dr Leora FoxAugust 08, 2022

Updates from uniQure about their gene therapy for Huntington's disease

Updates from uniQure about their gene therapy for Huntington's disease

uniQure is conducting safety trials of the first gene therapy for HD. A recent press release provided a 1 year update on the first group of 10 people who underwent surgery to receive this experimental drug. Let’s talk more about what it means.

Dr Leora Fox and Dr Rachel HardingJuly 11, 2022