Sodium phenylbutyrate in huntington's disease : A dose-finding study
Identifieur interne : 001501 ( PascalFrancis/Corpus ); précédent : 001500; suivant : 001502Sodium phenylbutyrate in huntington's disease : A dose-finding study
Auteurs : Penelope Hogarth ; Luca Lovrecic ; Dimitri KraincSource :
- Movement disorders [ 0885-3185 ] ; 2007.
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- Pascal (Inist)
English descriptors
- KwdEn :
Abstract
Transcriptional dysregulation in Huntington's disease (HD) is mediated in part by aberrant patterns of histone acetylation. We performed a dose-finding study in human HD of sodium phenylbutyrate (SPB), a histone deacetylase inhibitor that ameliorates the HD phenotype in animal models. We used a dose-escalation/de-escalation design, using prespecified toxicity criteria and standard clinical and laboratory safety measures. The maximum tolerated dose was 15 g/day. At higher doses, toxicity included vomiting, lightheadedness, confusion, and gait instability. We saw no significant laboratory or electrocardiographic abnormalities. Gene expression changes in blood suggested an inverse dose-response. In conclusion, SPB at 12 to 15 g/day appears to be safe and well-tolerated in human HD.
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Format Inist (serveur)
NO : | PASCAL 08-0015800 INIST |
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ET : | Sodium phenylbutyrate in huntington's disease : A dose-finding study |
AU : | HOGARTH (Penelope); LOVRECIC (Luca); KRAINC (Dimitri) |
AF : | Department of Neurology, Oregon Health & Science University/Portland, Oregon/Etats-Unis (1 aut.); Parkinson's Disease Research, Education & Clinical Center, Portland VA Medical Cente r/Portland, Oregon/Etats-Unis (1 aut.); Department of Neurology, Massachusetts General Hospital, Harvard Medical School, MassGeneral Institute for Neurodegeneration (MIND)/Charlestown, Massachusetts/Etats-Unis (2 aut., 3 aut.) |
DT : | Publication en série; Courte communication, note brève; Niveau analytique |
SO : | Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2007; Vol. 22; No. 13; Pp. 1962-1964; Bibl. 8 ref. |
LA : | Anglais |
EA : | Transcriptional dysregulation in Huntington's disease (HD) is mediated in part by aberrant patterns of histone acetylation. We performed a dose-finding study in human HD of sodium phenylbutyrate (SPB), a histone deacetylase inhibitor that ameliorates the HD phenotype in animal models. We used a dose-escalation/de-escalation design, using prespecified toxicity criteria and standard clinical and laboratory safety measures. The maximum tolerated dose was 15 g/day. At higher doses, toxicity included vomiting, lightheadedness, confusion, and gait instability. We saw no significant laboratory or electrocardiographic abnormalities. Gene expression changes in blood suggested an inverse dose-response. In conclusion, SPB at 12 to 15 g/day appears to be safe and well-tolerated in human HD. |
CC : | 002B17; 002B17G; 002B17A03 |
FD : | Pathologie du système nerveux; Chorée de Huntington; Sodium |
FG : | Pathologie de l'encéphale; Syndrome extrapyramidal; Maladie dégénérative; Maladie héréditaire; Pathologie du système nerveux central |
ED : | Nervous system diseases; Huntington disease; Sodium |
EG : | Cerebral disorder; Extrapyramidal syndrome; Degenerative disease; Genetic disease; Central nervous system disease |
SD : | Sistema nervioso patología; Corea Huntington; Sodio |
LO : | INIST-20953.354000173554140190 |
ID : | 08-0015800 |
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Pascal:08-0015800Le document en format XML
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<front><div type="abstract" xml:lang="en">Transcriptional dysregulation in Huntington's disease (HD) is mediated in part by aberrant patterns of histone acetylation. We performed a dose-finding study in human HD of sodium phenylbutyrate (SPB), a histone deacetylase inhibitor that ameliorates the HD phenotype in animal models. We used a dose-escalation/de-escalation design, using prespecified toxicity criteria and standard clinical and laboratory safety measures. The maximum tolerated dose was 15 g/day. At higher doses, toxicity included vomiting, lightheadedness, confusion, and gait instability. We saw no significant laboratory or electrocardiographic abnormalities. Gene expression changes in blood suggested an inverse dose-response. In conclusion, SPB at 12 to 15 g/day appears to be safe and well-tolerated in human HD.</div>
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<ET>Sodium phenylbutyrate in huntington's disease : A dose-finding study</ET>
<AU>HOGARTH (Penelope); LOVRECIC (Luca); KRAINC (Dimitri)</AU>
<AF>Department of Neurology, Oregon Health & Science University/Portland, Oregon/Etats-Unis (1 aut.); Parkinson's Disease Research, Education & Clinical Center, Portland VA Medical Cente r/Portland, Oregon/Etats-Unis (1 aut.); Department of Neurology, Massachusetts General Hospital, Harvard Medical School, MassGeneral Institute for Neurodegeneration (MIND)/Charlestown, Massachusetts/Etats-Unis (2 aut., 3 aut.)</AF>
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