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…

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…

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.…

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…

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…

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…