Articles with the topic: disease-modifying

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

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.

Dr Sarah HernandezJuly 11, 2024

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 Roche and Ionis - ASO Trial Halted Early

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

Dr Jeff Carroll and Dr Rachel HardingMarch 23, 2021

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

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

Dr Jeff CarrollJanuary 30, 2019

Advances on many fronts in the battle against the protein that causes Huntington's disease

Advances on many fronts in the battle against the protein that causes Huntington's disease

This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager

Dr Jeff CarrollDecember 04, 2018

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Has a "wonder drug" for dementia been discovered? (Spoiler alert: no.)

Media reports of a wonder drug for neurodegenerative diseases like Huntington's disease are overhyped

Dr Michael FlowerMay 07, 2017

Important advances in next generation genome editing tools for Huntington's Disease

Important advances in next generation genome editing tools for Huntington's Disease

Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials

Mr. Shawn MinnigOctober 31, 2016

Liftoff: First humans treated with gene silencing drugs for HD!

Liftoff: First humans treated with gene silencing drugs for HD!

Major Announcement: First HD patients treated with gene silencing drugs

Dr Jeff CarrollOctober 22, 2015

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