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Huntington's Month in Argentina - and a concert to celebrate it!

Claudia has managed to stage an impressive array of meetings during the month of May to celebrate the people affected by Huntington’s disease in Argentina. Part of this calendar of activities include a symposium to expose the social and medical issues affecting people suffering from HD. Both Claudia and I,…

Brenda's story

Brenda’s Story – as told by Claudia Perandones from Buenos Aires, Argentina I have a patient, called Daniel L., with Huntington’s disease, who is in a delicate condition because he can no longer move by himself and has to be fed by means of a gastrostomy tube. Daniel has a…

Viruses and the brain

Viruses and the brain JAN25 2015 I am certain most of you would have heard that some of the therapies being developed for HD and other neurodegenerative indications involve the use of viruses to deliver a gene therapy agent. This is true in the case of HD, we are working…

Clothes get delivered to communities in need - thank you friends for supporting Factor-H efforts

Finally, we were able to donate more than 500 pounds of clothes for the Colombian communities. DHL sent the boxes free of charge to Factor-H, courtesy of Conexion Colombia, an NGO who financed the shipment. The clothes were sent to El Dificil (Magdalena State), Juan de Acosta, Bogota, Medellin, and…

Visit to Feira Grande

We just visited the Brazilian community in Feira Grande, in northern Brazil.

Pfizer’s PDE10 inhibitor enters clinical trials for HD

Pfizer’s PDE10 inhibitor enters clinical trials for HD JUN16 2013 This is a good year for HD, and for all working to develop medicines to treat all those affected. For the first time in CHDI’s existence, a clinical trial with a new drug entity will start this month. The collaboration…

The importance of biomarkers

As I have described before, the age of molecular therapies for the treatment of HD is around the corner. But a critical component of the drug development process is still lacking: the identification of Htt-dependent biomarkers. Why is this important? Imagine what will happen over the next 1-3 years. Several…

Molecular therapies for HD – Ready, Set and GO!

Huntingtin targeting therapeutics are entering the clinical realm… after many years of work, 4 big players in the biotech/pharmaceutical industry are planning clinical trials in HD. Recently you might have heard about Roche, the Swiss pharmaceutical giant, acquiring the rights to develop ISIS’s lead antisense molecule to lower HTT expression…

Colombia Areas

Affected communities • Magdalena (El Difícil y San Ángel) • Juan de Acosta • Antioquía • Chocó • Bogotá Participating physicians • Dra. Natalia Costa Baena • Dra. Sonia Moreno • Dr. Oscar Bernal • Dr. Guillermo A. Monsalve Duarte • Dr. Mike Espinoza Participating community advocates Asociación Colombianos por…

Evolution, Huntingtin and Cancer?

A recent manuscript by a Swedish group (Ji et al., The Lancet, 2012) has revealed that the incidence of cancers amongst individuals bearing mutant expanded polyglutamine genes (and particularly Huntingtin expansions) is significantly lower than in the general population, even after adjusting for age. This study is a continuation of…
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