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Seminario del Dr. Muñoz-Sanjuán en la reunión de la sociedad de Neurología venezolana

Seminario del Dr. Muñoz-Sanjuán en la reunión de la sociedad de Neurología venezolana

Seminario del Dr. Ignacio Muñoz-Sanjuan, fundador de Factor-h, en la reunión anual de la sociedad venezolana de neurología, el 3 de junio 2023. Ignacio habla sobre programas terapéuticos actuales para la enfermedad de Huntington, comentando información actual de todos los programas sintomáticos y aquellos dirigidos a disminuir la expresión de…
Colombian Media covers the 2022 Latin American HD Congress in Barranquilla

Colombian Media covers the 2022 Latin American HD Congress in Barranquilla

News Papers like El Heraldo, with a daily circulation of 200.000 copies and around 1 million daily visits to its website, or Radio Caracol, the main national radio station of Colombia covered, among others, the 2022 Huntington Latin American Congress organized by Factor-H. El Heraldo headline Estigma social, el verdadero…
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…
CHDI's conference (Session II) - Seminars by Drs. José Lucas and Michael Rape

CHDI's conference (Session II) - Seminars by Drs. José Lucas and Michael Rape

Dr. José Lucas from the Center for Molecular Biology Severo Ochoa in Madrid spoke about this recent work in the role of mHTT in altered splicing and adenylation of mRNAs in HD. Jose started by describing his pioneering work in tau splicing alterations in the HD brain. He also described…
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