Yesterday, Uniqure reported on the 24-month results of their Phase 1/2 clinical trial with AMT-130, a viral gene therapy trial for HTT lowering for the treatment of HD. See the links to the press release and investor call slides. I wanted to review some aspects of the claims made by Uniqure, and…

From a gene mutation to a lethal disease HD is caused by a single mutation in a region of the HTT gene, located at the beginning of the gene (in exon 1), which contains a repeat sequence comprised of CAG repeats; in the normal range, people have between 17-22 repeats…

Gratitude day 2024 brings hope to Venezuela The importance of the contributions of the Venezuelan families to HD research and disease understanding cannot be minimized, even with the passing of time. The journey of these families in advancing our scientific and medical work started in the 1970s, after the presentation…

In the recent EHDN Newsletter, Factor-H work is featured in two articles – the first launches the 2024 Gratitude Day campaign; the second is by Alex Fisher, who tells her story of coming to Venezuela and the work she is conducting together with Factor-H & Habitat LUZ in Maracaibo to…

Today, November 29th is #givingtuesday, please think about people with Huntington in Latin América when giving your donations! Remember, GivingTuesday is a global generosity movement that unleashes the power of radical generosity around the world. Millions of people come together to celebrate generosity, share kindness, and drive record-breaking giving again…

Primero una Web, ahora una nueva forma de comunicar y colaborar… Son algo más de las 7 de la mañana en este pequeño y bellísimo pueblo de la Costa Brava donde vivo. Para quienes hayan oido hablar de él, está en Gerona en España y tiene una pequeña muralla alrededor…

A non-for-profit humanitarian project to increase the awareness of people living with and affected by Huntington’s disease, to facilitate social aid to diminish the suffering of local communities in Latin America. An effort to unite socially minded individuals through creative artistic, journalistic, medical, scientific, and political means to change the…

2020 was a year like no other. The global Covid-19 pandemic impacted the entire world, but it has taken a heavy toll on poor communities around the world. Most of the families we assist live in vulnerable conditions, and their access to employment and medical assistance were significantly impacted.  We reacted…

En las últimas semanas ha habido algunas noticias en la prensa sobre la progresión en fases clínicas de varios agentes potencialmente terapéuticos para tratar la enfermedad de Huntington. Estos avances continúan llamando la atención general y de los especialistas médicos, por el sencillo hecho de que este tipo de terapias…

My first – unexpected – science lecture for medical students in Barranquilla, Colombia Barranquilla is close to Juan de Acosta, the second largest cluster of HD patients in the world (to our knowledge). The prevalence was reported quite a few years ago by Dr. Daza in one of the few…