Articles with the topic: drug-development

Huntington’s disease clinical trial round up

Huntington’s disease clinical trial round up

Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease

Dr Rachel HardingApril 26, 2021

Good news from uniQure: gene therapy trial on track, and promising data in animals

Good news from uniQure: gene therapy trial on track, and promising data in animals

The first group of 10 participants have been dosed in uniQure’s clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.

Dr Leora FoxApril 15, 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

Uncovering the dark side of DNA repair to design HD treatments

Uncovering the dark side of DNA repair to design HD treatments

A gene called MSH3 helps to repair our DNA, but in HD it can slip up and cause CAG repeats to lengthen. Researchers have uncovered new information about how MSH3 activity is controlled, opening the door to new therapeutic avenues.

Emma BuntingDecember 22, 2020

Huntington Study Group (HSG) 2020 Annual Conference: HD in Focus - Day 1

Huntington Study Group (HSG) 2020 Annual Conference: HD in Focus - Day 1

Read our breakdown of some of the talks and presentations at day 1 of the Huntington Study Group (HSG) 2020 annual conference: HD in focus

Dr Rachel HardingOctober 30, 2020

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

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