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Latest news on Friedreich's Ataxia research
Updated results from clinical trials and research initiatives
Friedreich's Ataxia fundraising activities and staying in touch
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Thanks for the news and research items you send us - I would never be able to stay on top of all the developments if it weren't for you!
This is wonderful news, Cornelia
----- Original Message -----
Sent: Friday, September 05, 2008 11:56 AM
Subject: [FAPG] ENG: MDA press release- MDA Awards $1 Million to Repligen for Drug Development in Friedreich's Ataxia
See also Repligen's press release
TUCSON, Ariz., Sept. 4, 2008 - The Muscular Dystrophy Association (MDA) announced today that it has awarded approximately $1 million to Repligen Corp. of Waltham, Mass., for development of new compounds to treat Friedreich's ataxia (FA).
The compounds under development are histone deacetylase (HDAC) inhibitors, a class of chemical compounds that change the way cells interpret genetic information, usually activating genes that would otherwise be silent. When genes are activated, they serve as instruction manuals for production of proteins.
In FA, a mutation in the gene for the frataxin protein results in greatly reduced levels of frataxin and severe neurologic and cardiac problems. Frataxin is required to regulate cellular iron levels, which can accumulate to toxic levels without it. HDAC inhibitors may activate silent frataxin genes and restore frataxin protein production and iron regulation.
"We're very pleased to be giving Repligen this award," said Sharon Hesterlee, MDA's Vice President for Translational Research. "It's a great example of what translational research is all about - moving basic science findings from academic laboratories into drug development settings."
MDA is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. Friederich's ataxia is one of the diseases covered by the Association. To find out more about MDA, call (800) 5... , or visit www.mda.org.
Repligen Corporation is a biopharmaceutical company focused on the development of therapeutics for neurological disorders. A number of drug development programs are currently being conducted to evaluate drug candidates in diseases such as bipolar disorder and Friedreich's ataxia. For more information about Repligen, visit www.repligen.com.
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If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
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.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
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.