CHDI conference session V - updates on the DBS clinical trial

CHDI conference session V - updates on the DBS clinical trial

Jan Vesper, Dusseldorf University Jan Vesper spoke on the status update of the recently completed European randomized-controlled DBS trial of the globus pallidus (GP) for the treatment of HD (HD-DBS study). Prior work done in a pilot study in 2014 showed a significant reduction in chorea scores of the UHDRS.…
CHDI conference session V - clinical updates from Uniqure AMT-130 program

CHDI conference session V - clinical updates from Uniqure AMT-130 program

David Cooper David Cooper from Uniqure spoke about the current status and future plan for the first gene therapy trial for HD involving an AAV5 expressing a microRNA targeting HTT human exon-1 sequences, termed AMT-130. David described the preclinical work conducted to support the clinical trial data, in work that…
CHDI Conference Session V - Clinical trial updates

CHDI Conference Session V - Clinical trial updates

Drs. Lauren Boak and Peter McColgan (Roche) Drs. Lauren Boak and Peter McColgan discussed additional results from the tominersen Generation-HD1 phase III trial. Lauren spoke about the on-treatment and post-treatment effects of tominersen, both during the Phase III study and other studies involving this drug, with the focus of dose…
CHDI Conference Session IV - Drs. Peter McColgan & Jonas Dorn (Roche), Sarah Tabrizi & Jeff Long

CHDI Conference Session IV - Drs. Peter McColgan & Jonas Dorn (Roche), Sarah Tabrizi & Jeff Long

Peter McColgan & Jonas Dorn Peter McColgan from Roche spoke about the digital monitoring of cognitive and motor symptoms from the longitudinal study Generation HD1 in the tominersen trials. Peter described the digital monitoring platform employed during this therapeutic study (n=798 individuals). The platform was also used in the natural…
CHDI Conference Session IV - Drs. Jim Rosinski and Aline Delva

CHDI Conference Session IV - Drs. Jim Rosinski and Aline Delva

Jim Rosinski Jim Rosinski from the CHDI Foundation spoke about the -omics strategy at CHDI Foundation to identify early biomarkers of disease progression, with a focus today on the early analysis of the CSF and plasma samples derived from the HD Clarity study using the Somalogics platform. The goal of…
Session III CHDI Conference: Drs. Beth Stevens and Leslie Thompson

Session III CHDI Conference: Drs. Beth Stevens and Leslie Thompson

Beth Stevens Beth Stevens from HHMI and the Broad Institute spoke about her work in protecting synapses in the context of HD and the role of the immune system and microglial cells of the brain in modulating synaptic pruning in the adult brain, which enables the ‘sculpting’ of synaptic circuits.…
Session III CHDI Conference: Drs. Gene Yeo and Irina Antonijevic

Session III CHDI Conference: Drs. Gene Yeo and Irina Antonijevic

Dr. Gene Yeo from UCSD spoke about his recent work on targeting RNA degradation using Crispr-Cas13d. His lab focused on RNA binding proteins (RBPs) as regulators of gene expression, and as drug targets. He has developed methods to systematically study RBP functions, such as the enhanced eCLIP method, which his…
Colombian HD families housing improvement project

Colombian HD families housing improvement project

Factor-H continues to deliver on its promises. We try to provide opportunities for families affected by HD. Sometimes, their needs require home improvement projects – they simply need nothing more than a better home to care for their family members. Slowly, one by one, Factor-H is raising the funds necessary…
2021 Course on targeting mutant HTT therapeutically for clinicians of the movement disorder society (MDS) by Ignacio Munoz-Sanjuan

2021 Course on targeting mutant HTT therapeutically for clinicians of the movement disorder society (MDS) by Ignacio Munoz-Sanjuan

In this 2021 course, Dr. Ignacio Munoz-Sanjuan, VP of translational biology at CHDI Foundation, speaks to the clinicians and movement disorder residents as part of a course about Huntington’s disease organized by EHDN and MDS. The seminar focuses on our current understanding of HTT biological aspects relevant to the development…
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