Articles with the topic: huntingtin-lowering

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

Disappointing Results from Wave’s PRECISION-HD1 and 2 Trials

Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.

Dr Rachel Harding and Dr Leora FoxMarch 30, 2021

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

GPR52: Exploring a new way to lower huntingtin

GPR52: Exploring a new way to lower huntingtin

A Chinese research team developed a new way to lower huntingtin protein indirectly, by targeting a protein called GPR52. The molecules they designed were protective in cells and in mice with HD.

Dr Rachel HardingFebruary 11, 2021

Treatment for neurological disorder could be repurposed for Huntington’s disease patients

Treatment for neurological disorder could be repurposed for Huntington’s disease patients

While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation.

Dr Rachel HardingOctober 22, 2020

What does COVID-19 mean for Huntington’s disease families and HD research?

What does COVID-19 mean for Huntington’s disease families and HD research?

COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?

Dr Sarah Hernandez and Professor Ed WildApril 06, 2020

Huntington’s disease therapeutics conference 2020 - Day 3

Huntington’s disease therapeutics conference 2020 - Day 3

HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs

Dr Sarah Hernandez, Dr Rachel Harding, and Joel StantonMarch 03, 2020

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement

Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?

Dr Jeff CarrollJanuary 03, 2020

Could molecular handcuffs lower the protein that causes Huntington's disease?

Could molecular handcuffs lower the protein that causes Huntington's disease?

Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine

Dr Tamara MaiuriNovember 12, 2019

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin lowering tool described

Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.

Dr Jeff CarrollAugust 05, 2019