Original Message -----
From: Marilyn Downing
Sent: Tuesday, July 01, 2008 12:37 AM
Subject: [FAPG] Update on clinical research

Hello all,
Many thanks to Paul for his thorough posts with info about clinical trials, eligibility stats, NIH and FDA procedures etc. 
To add to that, I've asked Jennifer Farmer, FARA's Executive Director, to give us an update on the latest and this was her response:


Here are some specific updates on the trials:

Idebenone – Santhera has increased recruitment goal to 70 and they need about 7 more people, specifically at the UCLA site. FARA just sent out a second recruitment notice on through the registry.

Deferiprone – Phase II study has started at two sites in Europe. Apopharma is working on additional sites in Europe, one Canadian site and one Australian site.

A0001 – Penwest has submitted the Phase I study to the FDA and if approved the study will begin in July. Of note, this is a Phase I, dose-escalation study in healthy subjects.

EPO – Edison in collaboration with Dr. Martin Delatycki (Melbourne, Australia) and Dr. Bernard Ravina (University of Rochester) are preparing to meet with the FDA to discuss their EPO protocol for a clinical trial. The goal is to launch a trial by the end of the year.


Of note, besides funding and supporting these clinical trials, FARA continues to fund basic and translational research in an attempt to "have all the bases covered". 


Marilyn Downing
Secretary, Board of Directors
Grassroots Fundraising
Friedreich's Ataxia Research Alliance (FARA)
PO Box 1537
Springfield, VA 22151 USA
               (703) 426-1576        
email  marilyn.downing@...

Please register in the FARA Patient Registry at www.cureFA.org/registry

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!



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:


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.



Go to top