The hidden work of Factor-H - the story of Nelson

The hidden work of Factor-H - the story of Nelson

Nelson Echeverría is 49 years old and he is one of the HD individuals we assist. He is a wonderful person and always receives our social workers with a big smile. He lives in the outskirts of the town of Saco in the Atlántico department in Northern Colombia. He lives…
Actress Carmen Navarro, "La Conejo" in the TV show La Reina del Sur, gives explicit support to families with HD in Latin America

Actress Carmen Navarro, "La Conejo" in the TV show La Reina del Sur, gives explicit support to families with HD in Latin America

Actress Carmen Navarro, "La Conejo" in the TV show La Reina del Sur, gives explicit support to families with HD in Latin America
Visiting Colombia after the pandemic

Visiting Colombia after the pandemic

Finally the Factor-H team got together in Colombia in April 2022, after a hiatus of two and a half years. During the trip, Ignacio Muñoz and Roger Cachope went to visit the families and kids we support on the Caribbean states, and spent 8 days traveling from town to town…
Here is how you too can help! Thanks Ivan Angulo!

Here is how you too can help! Thanks Ivan Angulo!

Everyone can make a contribution to help those most affected by Huntington’s disease. In this brief post, we wanted to thank Dr. Iván Angulo for his commitment to rare disorder research. Iván is a former colleague of Nacho who reached out to raise funds for our Venezuelan colleagues suffering from…
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…
Sessão III CHDI Conferência: Drs. Beth Stevens e Leslie Thompson

Sessão III CHDI Conferência: Drs. Beth Stevens e 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.…
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