Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.
Identifieur interne : 000E25 ( Main/Merge ); précédent : 000E24; suivant : 000E26Erythropoietin 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
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pubmed:22411849Le document en format XML
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<author><name sortKey="Lauria, Giuseppe" sort="Lauria, Giuseppe" uniqKey="Lauria G" first="Giuseppe" last="Lauria">Giuseppe Lauria</name>
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<author><name sortKey="Cappellini, Maria D" sort="Cappellini, Maria D" uniqKey="Cappellini M" first="Maria D" last="Cappellini">Maria D. Cappellini</name>
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<series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term>
<term>Antioxidants (therapeutic use)</term>
<term>Dose-Response Relationship, Drug</term>
<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>
<term>Male</term>
<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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<keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Antioxidants</term>
<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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<keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Friedreich Ataxia</term>
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<keywords scheme="MESH" xml:lang="en"><term>Adult</term>
<term>Dose-Response Relationship, Drug</term>
<term>Double-Blind Method</term>
<term>Female</term>
<term>Humans</term>
<term>Male</term>
<term>Statistics, Nonparametric</term>
<term>Time Factors</term>
<term>Treatment Outcome</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>
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