Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.
Identifieur interne : 000D98 ( Main/Exploration ); précédent : 000D97; suivant : 000D99Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.
Auteurs : Caterina Mariotti [Italie] ; Roberto Fancellu ; Serena Caldarazzo ; Lorenzo Nanetti ; Daniela Di Bella ; Massimo Plumari ; Giuseppe Lauria ; Maria D. Cappellini ; Lorena Duca ; Alessandra Solari ; Franco TaroniSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2012.
English descriptors
- KwdEn :
- Adult, Antioxidants (therapeutic use), Dose-Response Relationship, Drug, Double-Blind Method, Erythropoietin (therapeutic use), Female, Friedreich Ataxia (drug therapy), Friedreich Ataxia (metabolism), Humans, Iron-Binding Proteins (metabolism), Male, Recombinant Proteins (therapeutic use), Statistics, Nonparametric, Time Factors, Treatment Outcome, Ubiquinone (analogs & derivatives), Ubiquinone (therapeutic use), Young Adult.
- MESH :
- chemical , analogs & derivatives : Ubiquinone.
- chemical , metabolism : Iron-Binding Proteins.
- chemical , therapeutic use : Antioxidants, Erythropoietin, Recombinant Proteins, Ubiquinone.
- drug therapy : Friedreich Ataxia.
- metabolism : Friedreich Ataxia.
- Adult, Dose-Response Relationship, Drug, Double-Blind Method, Female, Humans, Male, Statistics, Nonparametric, Time Factors, Treatment Outcome, Young Adult.
Abstract
Friedreich ataxia is a rare disease caused by GAA-trinucleotide-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 with Friedreich ataxia.
DOI: 10.1002/mds.24066
PubMed: 22411849
Affiliations:
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Le document en format XML
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<author><name sortKey="Nanetti, Lorenzo" sort="Nanetti, Lorenzo" uniqKey="Nanetti L" first="Lorenzo" last="Nanetti">Lorenzo Nanetti</name>
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<author><name sortKey="Di Bella, Daniela" sort="Di Bella, Daniela" uniqKey="Di Bella D" first="Daniela" last="Di Bella">Daniela Di Bella</name>
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<author><name sortKey="Duca, Lorena" sort="Duca, Lorena" uniqKey="Duca L" first="Lorena" last="Duca">Lorena Duca</name>
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<author><name sortKey="Solari, Alessandra" sort="Solari, Alessandra" uniqKey="Solari A" first="Alessandra" last="Solari">Alessandra Solari</name>
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<series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title>
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<term>Double-Blind Method</term>
<term>Erythropoietin (therapeutic use)</term>
<term>Female</term>
<term>Friedreich Ataxia (drug therapy)</term>
<term>Friedreich Ataxia (metabolism)</term>
<term>Humans</term>
<term>Iron-Binding Proteins (metabolism)</term>
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<term>Recombinant Proteins (therapeutic use)</term>
<term>Statistics, Nonparametric</term>
<term>Time Factors</term>
<term>Treatment Outcome</term>
<term>Ubiquinone (analogs & derivatives)</term>
<term>Ubiquinone (therapeutic use)</term>
<term>Young Adult</term>
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<term>Erythropoietin</term>
<term>Recombinant Proteins</term>
<term>Ubiquinone</term>
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<keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Friedreich Ataxia</term>
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<term>Dose-Response Relationship, Drug</term>
<term>Double-Blind Method</term>
<term>Female</term>
<term>Humans</term>
<term>Male</term>
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<front><div type="abstract" xml:lang="en">Friedreich ataxia is a rare disease caused by GAA-trinucleotide-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 with Friedreich ataxia.</div>
</front>
</TEI>
<affiliations><list><country><li>Italie</li>
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<region><li>Lombardie</li>
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<settlement><li>Milan</li>
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<tree><noCountry><name sortKey="Caldarazzo, Serena" sort="Caldarazzo, Serena" uniqKey="Caldarazzo S" first="Serena" last="Caldarazzo">Serena Caldarazzo</name>
<name sortKey="Cappellini, Maria D" sort="Cappellini, Maria D" uniqKey="Cappellini M" first="Maria D" last="Cappellini">Maria D. Cappellini</name>
<name sortKey="Di Bella, Daniela" sort="Di Bella, Daniela" uniqKey="Di Bella D" first="Daniela" last="Di Bella">Daniela Di Bella</name>
<name sortKey="Duca, Lorena" sort="Duca, Lorena" uniqKey="Duca L" first="Lorena" last="Duca">Lorena Duca</name>
<name sortKey="Fancellu, Roberto" sort="Fancellu, Roberto" uniqKey="Fancellu R" first="Roberto" last="Fancellu">Roberto Fancellu</name>
<name sortKey="Lauria, Giuseppe" sort="Lauria, Giuseppe" uniqKey="Lauria G" first="Giuseppe" last="Lauria">Giuseppe Lauria</name>
<name sortKey="Nanetti, Lorenzo" sort="Nanetti, Lorenzo" uniqKey="Nanetti L" first="Lorenzo" last="Nanetti">Lorenzo Nanetti</name>
<name sortKey="Plumari, Massimo" sort="Plumari, Massimo" uniqKey="Plumari M" first="Massimo" last="Plumari">Massimo Plumari</name>
<name sortKey="Solari, Alessandra" sort="Solari, Alessandra" uniqKey="Solari A" first="Alessandra" last="Solari">Alessandra Solari</name>
<name sortKey="Taroni, Franco" sort="Taroni, Franco" uniqKey="Taroni F" first="Franco" last="Taroni">Franco Taroni</name>
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<country name="Italie"><region name="Lombardie"><name sortKey="Mariotti, Caterina" sort="Mariotti, Caterina" uniqKey="Mariotti C" first="Caterina" last="Mariotti">Caterina Mariotti</name>
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