Thousands Suffer From Friedreich's Ataxia

(News kindly supplied by Michel Beaudet through Internaf)


BOSTON, September 4, 2009 -- MDA has led the world in neuromuscular disease research for nearly 60 years. In that time, scientific technology has grown more costly and more complex. To share in the costs, MDA has formed partnerships with many biotechnology companies. A Massachusetts company is working to find a cure for Friedreich's Ataxia. "Repligen is working to develop a novel therapy for treatment of Friedreich's Ataxia. And if we are successful in developing these drugs, it has a potential to be the first drug available and it might modify the course of the disease for the patients and significantly improve the course and trajectory of their life," said Laura Whitehouse, of Repligen. Friedreich's Ataxia is an inherited neurodegenerative disease that is characterized by low levels of the protein Frataxin, and it leads to progressive disability. It affects the nervous system and muscle function. "At Repligen, my responsibility is to develop some methods that we can use to monitor how well our compounds do at increasing the frataxin gene which is the gene that is under functioning in Friedreich's Ataxia," said scientist Heather Plasterer. "We are in the process of developing a drug that we think is actually going to modify FA not just treat the symptoms, but reverse the symptoms. And we are pretty enthusiastic excited about that," Repligen's Steve Jones said.


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The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

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!

https://www.amazon.com/Legacy-Marie-Schlau-collective-Friedreichs-ebook/dp/B01N28AFWZ

 

Research projects currently being financed by BabelFAmily

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:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

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:

https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
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.

 

 

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