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Terapias moleculares para la Enfermedad de Huntington: últimas noticias

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…

Wave Life Sciences empieza estudios clínicos con un ASO que degrada la proteína mutante

La última vez que escribí comenté sobre la gran expectativa que tenemos todos sobre los estudios a comenzar de fase 3 con una molécula de antisense (ASO) de la compañia Ionis con Roche. Los estudios de fase 2 habían concluído positivamente, la droga era bien tolerada, y se veían claros…

Noticias sobre el estudio de Ionis/Roche con oligonucleótidos para el tratamiento de Huntington

Recientemente ha habido mucho interés en los estudios realizados por la compañia de California Ionis, en colaboración con la farmacéutica Roche de Suiza, en pacientes de Huntington. Su droga, un compuesto nuevo llamado HTT-Rx, es una molécula de ADN de la clase de fármacos llamados oligonucleótidos antisentidos, debido al hecho…
A letter from a scientist

A letter from a scientist

I am enclosing here a piece I wrote during a lengthy period in 2015, which led to the publication in Nature of a short article about the issues surrounding the Latin American HD communities. I am attaching it in its entirety here. The Nature commentary was a shortened version of…
Meeting with Pope Francis at the Vatican May 18, 2017

Meeting with Pope Francis at the Vatican May 18, 2017

Factor-H is pleased to announce a private audience with Pope Francis at the Vatican on May 18, 2017 (11am) to honor Latin American families affected by HD
Roberto

Roberto

I met Roberto and his family when I visited them in April in Juan de Acosta, in the Atlántico State of the Colombian Caribbean coast. Juan de Acosta was considered the second largest HD cluster in the world, although many affected families have left the town in the recent years.…
New article about Colombia and Factor-H in the Lancet Neurology

New article about Colombia and Factor-H in the Lancet Neurology

Dara Mohammadi just wrote a final piece from the trip we took together to Colombia in April 2016. You can find the pdf of the article here: HD Colombia Lancet Also read about Enroll-HD efforts here: http://www.enroll-hd.org/ Please note that even though efforts to initiate Enroll-HD are underway in Colombia,…
Article by Monica Roa - a lawyer working with Factor-H to protect the rights of the patients in Colombia

Article by Monica Roa - a lawyer working with Factor-H to protect the rights of the patients in Colombia

A couple of months ago, I met Monica Roa in Washington DC to discus whether we could team up to deal with the issues facing many of the Colombian families – the lack of institutional and government support, the refusal by many of the insurance companies (called EPS in Colombia)…
Start of 'Proyecto Abrazos' - The Children's Project

Start of 'Proyecto Abrazos' - The Children's Project

Sábanas de San Angel – this past April. We pulled in with Fermín’s car loaded with food, hammocks, and clothes. The scenery was arid – this region has suffered from a profound drought for a while now, and everything is dried up. The Cañahuate trees the only bright color amidst…
Colombia April 2016 - Part 3: Stories from Juan de Acosta

Colombia April 2016 - Part 3: Stories from Juan de Acosta

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…
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