Articles with the topic: drug-development

Sad news from the SIGNAL study: pepinemab does not influence HD symptoms

Sad news from the SIGNAL study: pepinemab does not influence HD symptoms

The SIGNAL study did not meet its key clinical goals for #HuntingtonsDisease to slow or improve HD symptoms, but the results are still informative for the HD community and other fields.

Dr Jeff CarrollSeptember 23, 2020

When genes are unstable: targeting somatic instability in HD

When genes are unstable: targeting somatic instability in HD

CAG repeats expand in some parts of the body and brain as people with HD get older, a phenomenon known as somatic instability. Learn more about how researchers are exploring somatic instability and DNA repair to design therapies for HD.

Dr Rachel Harding and Dr Leora FoxSeptember 08, 2020

HD Young Adult Study defines the sweet spot: symptom-free with measurable changes

HD Young Adult Study defines the sweet spot: symptom-free with measurable changes

We know that HD-related changes can occur many years before symptom onset, but how early do those changes begin? A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults.

Dr Sarah HernandezMay 27, 2020

New molecule can reverse the Huntington's disease mutation in lab models

New molecule can reverse the Huntington's disease mutation in lab models

A collaborative team of scientists from Canada and Japan have identified a small molecule which can change the CAG-repeat length in different lab models of Huntington's disease. #HuntingtonsDisease #DrugDiscovery

Dr Michael FlowerApril 06, 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 Dr Ed WildApril 06, 2020

Huntington’s disease therapeutics conference 2020 - Day 1

Huntington’s disease therapeutics conference 2020 - Day 1

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

Dr Rachel Harding, Dr Sarah Hernandez, and Joel StantonMarch 12, 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