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…

In April 2014, the first home renovation project started in Medellin, as a collaborative project between Factor-H, Habitat for Humanity, and Acolpeh. Together we raised over US$8000 through the Habitat web site, and together we identified a few families in need of improved housing conditions. The first project took 3…

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…

Claudia Perandones put this video together for her recent visit to the CHDI offices in Los Angeles, USA. It is a moving account of all the clusters that are documented in latinamerica. Please look at it, share it, and help increase awareness of their existence and of the problems these communities…

Last week, CHDI hosted a stem cell meeting in Princeton, NJ, chaired by Thomas Vogt (CHDI’s new VP for Discovery & Systems Biology), and myself. We hosted several eminent scientists working on the use of stem cells for understanding HD pathogenesis, and identifying new mechanisms for potential therapeutic treatments. The…

500 pounds (300+ kilograms) of donated clothes have arrived at several communities in Colombia. Thanks to the Conexion Colombia NGO (www.conexioncolombia.com) , all the donated clothes Factor-H collected in Los Angeles have made it to their destinations. This is the first ‘international’ shipment of donated goods to benefit the Colombian…

Human genetics research sheds new hope in modulating the progression of HD MAR23 2014 It is not unfair to say that almost the entire field of therapeutic development for the treatment of HD is founded on the learnings that stem from the cloning of the gene causative of HD, the…

Last week, Claudia and I from Factor-H, together with the Venezuelan physicians and patient association (Avehun), we spent a day visiting the neighborhood of San Luis, outside Maracaibo. We hand-delivered clothes donated by our US friends and colleagues, to about 50 people living with HD in their families. It was…

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…

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…