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Friedreich's ataxia: clinical pilot trial with recombinant human erythropoietin

✍ Scribed by Sylvia Boesch; Brigitte Sturm; Sascha Hering; Hans Goldenberg; Werner Poewe; Barbara Scheiber-Mojdehkar

Publisher: John Wiley and Sons
Year: 2007
Tongue: English
Weight: 104 KB
Volume: 62
Category: Article
ISSN: 0364-5134
DOI: 10.1002/ana.21177

No coin nor oath required. For personal study only.

✦ Synopsis

Abstract

To determine the role of recombinant human erythropoietin as a possible treatment option in Friedreich's ataxia, we performed an open‐label clinical pilot study. Primary outcome measure was the change of frataxin levels at week 8 versus baseline. Twelve Friedreich's ataxia patients received 5,000 units recombinant human erythropoietin three times weekly subcutaneously. Frataxin levels were measured in isolated lymphocytes by enzyme‐linked immunosorbent assay. In addition, urinary 8‐hydroxydeoxyguanosine and serum peroxides, were measured. Treatment with recombinant human erythropoietin showed a persistent and significant increase in frataxin levels after 8 weeks (p < 0.01). All patients showed a reduction of oxidative stress markers. Ann Neurol 2007

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