At other times, stepping into the non-HD world allowed them to rest, recover, and reconnect with who they were, beyond the ...
This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial. The results of a clinical trial, called VIBRANT-HD, investigating an oral ...
The sequence pattern matters: Interruptions or “hitches” and loss of these within the C-A-G repeat and C-C-G repeat stretches play a major role as to when symptoms start with loss of interruptions ...
For many people raised in families affected by Huntington’s disease (HD), childhood isn’t just about scraped knees and schoolbooks. It can also mean living with uncertainty, emotional turbulence, and ...
Huntington’s disease (HD) is a fatal brain disorder caused by a defect in the huntingtin (HTT) gene, which produces an extra-long protein. This defect causes the HTT protein to form harmful clumps, or ...
INGREZZA is the trade name of valbenazine, a drug developed by the company Neurocrine Biosciences. It works similarly to tetrabenazine and deutetrabenazine (Austedo), drugs commonly prescribed to help ...
This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...
DNA-based drugs called antisense oligonucleotides, or ASOs, are now in multiple clinical trials in Huntington’s disease, aiming to lower production of the harmful mutant huntingtin protein in the ...
UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the ...
A 14-year study tracking NfL levels in people with the HD gene shows this tiny protein can signal disease progression many years before symptoms start. The longest follow-up study yet adds weight to ...
Huntington’s disease (HD) is caused by a single mutation in a gene we refer to as the HD gene. Like most genes, the HD gene is used by cells as instructions to create a little cellular machine called ...
Scientists searching for new ways to stop Huntington’s disease (HD) have focused in on the repeating C-A-G letters of genetic code that cause the disease. That’s because the exact way these C-A-G ...
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