Abstract
Nausea and dysautonomic crises severely limit function and quality of life for a large number of individuals with familial dysautonomia. We treated a small cohort of 15 patients with familial dysautonomia who suffered frequent dysautonomic crises with pregabalin. Nausea and overt crises markedly decreased in 13 (87%) of these patients and the overall assessments of benefit were extremely favorable, suggesting that pregabalin may be a potentially useful therapeutic agent for this disorder.
Publication types
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Research Support, Non-U.S. Gov't
MeSH terms
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Adolescent
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Adult
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Anticonvulsants / adverse effects
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Anticonvulsants / therapeutic use*
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Carrier Proteins / genetics
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Child
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Dose-Response Relationship, Drug
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Drug Administration Schedule
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Drug Therapy, Combination
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Dysautonomia, Familial / diagnosis
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Dysautonomia, Familial / drug therapy*
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Dysautonomia, Familial / genetics
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Female
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Humans
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Male
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Pregabalin
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Transcriptional Elongation Factors
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Young Adult
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gamma-Aminobutyric Acid / adverse effects
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gamma-Aminobutyric Acid / analogs & derivatives*
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gamma-Aminobutyric Acid / therapeutic use
Substances
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Anticonvulsants
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Carrier Proteins
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Elp1 protein, human
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Transcriptional Elongation Factors
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Pregabalin
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gamma-Aminobutyric Acid