Thirty years beyond discovery - Clinical trials in succinic semialdehyde dehydrogenase deficiency, a disorder of GABA metabolism

Journal of Inherited Metabolic Disease

Volumn 36, Issue 3, 2013, Pages 401-410

  Vogel, Kara R ^a   Pearl, Phillip L ^b   Theodore, William H ^c   McCarter, Robert C ^b   Jakobs, Cornelis ^d   Gibson, K Michael ^a  

^a WASHINGTON STATE UNIVERSITY   (United States)

^b GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

^c NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^d VU UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

3 AMINOPROPYL(DIETHOXYMETHYL)PHOSPHINIC ACID; 3 AMINOPROPYLBUTYLPHOSPHINIC ACID; 3 MERCAPTOPROPIONIC ACID; 4 AMINOBUTYRIC ACID; 4 HYDROXYBUTYRIC ACID; 6,7,8,9 TETRAHYDRO 5 HYDROXY 5H BENZOCYCLOHEPT 6 YLIDENE ACETIC ACID; CGP 35358; CYCLOSERINE; ETIRACETAM; FLUMAZENIL C 11; GABAERGIC RECEPTOR AFFECTING AGENT; HISTIDINE; HOMOCARNOSINE; LAMOTRIGINE; NCS 382; SUCCINATE SEMIALDEHYDE DEHYDROGENASE; TAURINE; TOPIRAMATE; UNCLASSIFIED DRUG; VIGABATRIN;

4 AMINOBUTYRIC ACID METABOLISM; CLINICAL TRIAL (TOPIC); COGNITION; CONVULSION; DOSE RESPONSE; DRUG DOSE TITRATION; DRUG MECHANISM; DRUG MEGADOSE; DRUG WITHDRAWAL; ELECTROENCEPHALOGRAM; EPILEPTIC STATE; HUMAN; INBORN ERROR OF METABOLISM; INSOMNIA; KETOGENIC DIET; MILD COGNITIVE IMPAIRMENT; NEUROPATHOLOGY; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PATHOPHYSIOLOGY; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); RETINA DISEASE; REVIEW; SUCCINIC SEMIALDEHYDE DEHYDROGENASE DEFICIENCY; TREATMENT OUTCOME;

AMINO ACID METABOLISM, INBORN ERRORS; ANIMALS; CLINICAL TRIALS AS TOPIC; GABA-B RECEPTOR ANTAGONISTS; GAMMA-AMINOBUTYRIC ACID; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; MICE; MODELS, BIOLOGICAL; SUCCINATE-SEMIALDEHYDE DEHYDROGENASE; TIME FACTORS;

EID: 84877771785     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-012-9499-5     Document Type: Review

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