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Article Date: 26 Sep 2009 - 2:00 PDT
Repligen Corporation (Nasdaq: RGEN) reported publication of research that identifies histone deacetylase 3 (HDAC 3) as an important enzyme target for therapeutic intervention in Friedreich's ataxia. These research findings confirm the drug target of the HDAC inhibitors that Repligen is currently developing for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. The study entitled "Chemical Probes Identify a Role for Histone Deacetylase 3 in Friedreich's Ataxia Gene Silencing" published today in the journal Chemistry & Biology (volume 16, 980-989, September 25, 2009) was conducted in collaboration with scientists at The Scripps Research Institute.
"Prior research indicated that HDAC enzymes play an important role in silencing the gene implicated in Friedreich's ataxia," stated Walther C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "Identification of the involvement of HDAC 3 is an important step in developing a specific drug for Friedreich's ataxia without the potential toxicities associated with broad-acting HDAC inhibitors. There are more than 15,000 patients worldwide with Friedreich's ataxia with no therapies available for treatment."
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The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
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Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.