----- Original Message ----- From: Jennifer Callaghan To: FARAA mailinglist Sent: Thursday, January 15, 2009 3:25 AM Subject: [fara] FW: Clinical Trial in Australia From: This email address is being protected from spambots. You need JavaScript enabled to view it. [mailto:This email address is being protected from spambots. You need JavaScript enabled to view it.] Sent: Wednesday, 14 January 2009 2:55 PM To: This email address is being protected from spambots. You need JavaScript enabled to view it. Subject: Clinical Trial in Australia A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia Official Title: A Six-Month Double-Blind, Randomized, Placebo-Controlled Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia Detailed Description: This will be a multi-centre, double-blind, randomized, placebo-controlled clinical trial. A total of 80 patients with Friedreich's ataxia will be enrolled. Eligible patients will receive deferiprone oral solution or placebo at a total daily dose of 20 mg/kg/day, 40 mg/kg/day or 60 mg/kg/day, divided into two-daily doses for 6 months. Eligibility: 1. Patient must have a diagnosis of Friedreich’s ataxia, with confirmed mutation (excludes point mutation) in the frataxin (FXN) gene and GAA repeats ™ 400 on the shorter allele. 2. Patients must be between the ages of 7 to 35 years. 3. For at least 1 month prior to start of treatment and during the study, patients cannot have idebenone, coenzyme Q10, vitamin C, vitamin E or other antioxidants as a supplement or as a drug therapy. 4. Patients must not have an iron deficiency. 5. Patient must not have received any investigational drug products within 30 days prior to enrollment into this study. 6. Patients must not have previously taken deferiprone. Contact: Dr. Martin Delatycki, Murdoch Children’s Research Institute, Victoria, Australia, This email address is being protected from spambots. You need JavaScript enabled to view it. Dian Shaw, ApoPharma, tel: 1-416-401-7283, This email address is being protected from spambots. You need JavaScript enabled to view it. Felicia DeRosa, FARA, tel: 1-484-875-3015, This email address is being protected from spambots. You need JavaScript enabled to view it. œœœœœœœ Babel note See also: http://health.groups.yahoo.com/group/FA_babelFAmily/message/2414 (ENG) http://health.groups.yahoo.com/group/FA_babelFAmily/message/1012 (ENG)

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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