disease-modifying

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