Here's an extension study report on a subset of 8 of the original Austrian
EPO study.

The sample numbers are too small and the article too brief to really add any
clarity or fresh insight on EPO as a treatment.

Paul

 Neurological effects of recombinant human erythropoietin in Friedreich's
 ataxia: A clinical pilot trial.

 Department of Neurology, Innsbruck Medical University, Innsbruck, Austria.

 In a "proof-of-concept" study, we demonstrated that recombinant human
 erythropoietin (rhuEPO) increases frataxin levels in Friedreich's ataxia
 (FRDA) patients. We now report a 6-month open-label clinical pilot study
 of
 safety and efficacy of rhuEPO treatment in FRDA. Eight adult FRDA patients
 received 2.000 IU rhuEPO thrice a week subcutaneously. Clinical outcome
 measures included Ataxia Rating Scales. Frataxin levels and indicators for
 oxidative stress were assessed. Hematological parameters were monitored
 biweekly. Scores in Ataxia Rating Scales such as FARS (P = 0.0063) and
 SARA
 (P = 0.0045) improved significantly. Frataxin levels increased (P = 0.017)
 while indicators of oxidative stress such as urine 8-OHdG (P = 0.012) and
 peroxide levels decreased (P = 0.028). Increases in hematocrit requiring
 phlebotomies occurred in 4 of 8 patients. In this explorative open-label
 clinical pilot study, we found an evidence for clinical improvement
 together
 with a persistent increase of frataxin levels and a reduction of oxidative
 stress parameters in patients with FRDA receiving chronic treatment with
 rhuEPO. Safety monitoring with regular blood cell counts and parameters of
 iron metabolism is a potential limitation of this approach. (c) 2008
Movement Disorder Society.
 PMID: 18759345 [PubMed - as supplied by publisher]

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.

 

 

Go to top