Dancing at the Vatican

The Dancing at The Vatican Documentary

With over 20,000 views, Dancing at the Vatican documentary has been a success, and we hope this film has inspired people. We remember with fondness the trip to the Vatican and remain thankful for the words and warmth Pope Francis delivered to the HD community. The documentary is subtitled in

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Greetings from a New Year

It is now 9:52pm in Los Angeles and it is officially my birthday in Spain. I was born at 8am on the 8th January of 1972 – 49 years ago today. As I write this, life is the USA is very complicated – we have had an attack on democratic

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Una pastilla para tratar la enfermedad de Huntington?

Si me hubiesen preguntado hace unos años que en el 2020 empezaríamos estudios clínicos con una terapia en pastilla que trataría la causa de la enfermedad, no me lo hubiera creído. Durante mucho tiempo, desde que empecé a trabajar como científico en la fundación CHDI, he trabajado mucho en el

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Meet Brayan – the video

A few months ago I wrote a story about meeting Brayan, a young kid at risk for HD from Barranquitas, Venezuela (see blog post “Reflections on instilling a sense of hope”). Together with colleagues Matt Rota (https://www.mattrotasart.com) and Nicanor Cardeñosa, we put together a narrated illustrated story of that encounter.

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Updates on the clinical studies targeting HTT by Uniqure: AMT-130

The field of therapeutic development targeting HTT is proceeding well. In addition to the ongoing efforts by Roche and Wave with antisense drugs targeting HTT, new approaches are making their way to the clinic. Uniqure just put out a press release communicating that their clinical trial -the first of its

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