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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.
July 11, 2022
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.
November 03, 2022
Roche Phase II GENERATION HD2 study underway
Roche released a community letter in early 2023, to share that their Phase II clinical trial to study the huntingtin-lowering drug, tominersen, is now underway. In this article, we summarise the latest news about this huntingtin-lowering drug.
February 14, 2023
Tipping the balance; new insights into HD genetic modifiers
A new study from researchers at Thomas Jefferson University delves into the details of how genetic modifiers of Huntington’s disease work.
September 01, 2023
Putting it in print: GENERATION HD1 study results published
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?
December 07, 2023
The director’s cut: how CAG repeats change the editing of genetic messages
Scientists in Massachusetts have recently advanced our understanding of how repetitive sequences in DNA can disrupt the creation and editing of genetic messenger molecules in cells, and how this could lead to the production of harmful proteins.
March 26, 2024
Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial
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
August 16, 2024
Steadying genetic stumble could help slow Huntington's disease
Using CRISPR technology, scientists uncovered genes that control C-A-G genetic stumbles in Huntington's disease
February 11, 2025
Successful gene therapy trial in Parkinson’s Disease gives hope for HD
Success for brain ‘gene therapy’ in Parkinson’s disease – good news for similar treatments being developed in HD
March 31, 2011
A back-to-front hidden message in the HD gene?
Unzip your genes! A backwards message, hidden in the 'backup DNA' of the Huntington's disease gene... what does it me
August 19, 2011
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