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Deferiprone in Friedreich ataxia: A 6-Month randomized controlled trial

โœ Scribed by Pandolfo, Massimo; Arpa, Javier; Delatycki, Martin B.; Le Quan Sang, Kim Hanh; Mariotti, Caterina; Munnich, Arnold; Sanz-Gallego, Irene; Tai, Geneieve; Tarnopolsky, Mark A.; Taroni, Franco; Spino, Michael; Tricta, Fernando

Book ID
126772339
Publisher
John Wiley and Sons
Year
2014
Tongue
English
Weight
895 KB
Volume
76
Category
Article
ISSN
0364-5134

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โœ Caterina Mariotti; Roberto Fancellu; Serena Caldarazzo; Lorenzo Nanetti; Daniela ๐Ÿ“‚ Article ๐Ÿ“… 2012 ๐Ÿ› John Wiley and Sons ๐ŸŒ English โš– 844 KB

## Abstract ## Background: Friedreich ataxia is a rare disease caused by GAAโ€trinucleotideโ€repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human recombinant erythropoietin was reported to increase frataxin levels in patients w