MovDisordV3, Main, Merge, bibRecord, 004047

Sodium phenylbutyrate in huntington's disease : A dose-finding study

Identifieur interne : 004047 ( Main/Merge ); précédent : 004046; suivant : 004048

Sodium phenylbutyrate in huntington's disease : A dose-finding study

Auteurs : Penelope Hogarth [États-Unis] ; Luca Lovrecic [États-Unis] ; Dimitri Krainc [États-Unis]

Source :

Movement disorders [ 0885-3185 ] ; 2007.

RBID : Pascal:08-0015800

Descripteurs français

Pascal (Inist)
- Pathologie du système nerveux, Chorée de Huntington, Sodium.
Wicri :
- topic : Sodium.

English descriptors

KwdEn :
- Huntington disease, Nervous system diseases, Sodium.

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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Pascal:08-0015800

Le document en format XML

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<affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Department of Neurology, Massachusetts General Hospital, Harvard Medical School, MassGeneral Institute for Neurodegeneration (MIND)</s1>
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<author><name sortKey="Krainc, Dimitri" sort="Krainc, Dimitri" uniqKey="Krainc D" first="Dimitri" last="Krainc">Dimitri Krainc</name>
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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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	Movement Disorders (revue)
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Movement Disorders (revue)

Sodium phenylbutyrate in huntington's disease : A dose-finding study

Sodium phenylbutyrate in huntington's disease : A dose-finding study

Source :

Descripteurs français

English descriptors

Abstract

Links toward previous steps (curation, corpus...)

Links to Exploration step

Le document en format XML

Pour manipuler ce document sous Unix (Dilib)

Pour mettre un lien sur cette page dans le réseau Wicri