Monday, May 26, 2008

UCLA seeks patients 8-to-17 with Friedreich’s Ataxia for new drug study

Friedreich’s Ataxia (FRDA) is an inherited disease that causes progressive damage to the nervous system. UCLA Researchers are seeking patients 8-to-17 years old who have been diagnosed with Friedreich’s Ataxia for a Phase III clinical research trial. The purpose of the trial is to examine the possible therapeutic effects of the drug idebenone (SNT-MC17) on the nervous system and heart.

Friedreich’s Ataxia (FRDA) is an extremely rare disease, with no approved medication, that typically begins in childhood or early adolescence with symptoms that are slow and progressive. These include muscle weakness in the arms and legs; loss of coordination, vision impairment, curvature of the spine (scoliosis), and various heart disorders including atrial fibrillation, and enlargement of the heart.

A Phase III trial is a clinical trial to show the effectiveness and safety of a drug. The Food & Drug Administration requests such trials before a drug can be submitted for regulatory review and market approval. Earlier studies have shown idebenone to be effective with minimal side effects. The purpose of this study is to further show idebenone’s clinical benefit on the nervous system and the hearts of patients with FRDA. The trial is being led by Dr. Susan Perlman, UCLA clinical professor of neurology.

Ambulatory Patients between the ages of 8-to-17 years-old are sought who have been diagnosed with FRDA. Participation in the study will require a nine month commitment. For six of those months participants will be treated with the medication or placebo (an inactive drug with no drug substance in it). Study volunteers will be assigned randomly to receive either one of the two drug doses or placebo. Santhera Pharmaceuticals, the developer of SNT-MC17/idebenone, will offer all patients that complete the study the opportunity to participate in a follow up study. In that study, all participants will receive the high-dose of SNT-MC17/idebenone for up to one year free of charge.

Patients participating in this current trial will be asked to visit UCLA
Monday, May 26, 2008
9:00 am
Organization:  UCLA Department of Neurology
Venue:  UCLA Department of Neurology
Westwood Plaza
Los Angeles, CA 90095
Phone:  3102068153

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.



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