disease-modifying

Additional Clarity: What We Know 4 Weeks After the uniQure News

We’re 4 weeks out from uniQure’s positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions from the Huntington’s disease community?

By Dr Sarah Hernandez

October 23, 2025

Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515

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.

By Dr Sarah Hernandez

July 11, 2024

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

By Dr Rachel Harding & Dr Sarah Hernandez

August 30, 2022

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.

By Dr Sarah Hernandez & Dr Rachel Harding

August 29, 2022

Sad News from Roche and Ionis – ASO Trial Halted Early

Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early

By Dr Jeff Carroll, PhD & Dr Rachel Harding

March 23, 2021

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients

By Dr Jeff Carroll, PhD

January 29, 2019