A Randomized Controlled Trial of SGS-742, a γ-aminobutyric acid B (GABA-B) Receptor Antagonist, for Succinic Semialdehyde Dehydrogenase Deficiency

We examined safety, tolerability, and efficacy of SGS-742, a γ-aminobutyric acid B (GABA-B) receptor antagonist, in patients with succinic semialdehyde dehydrogenase deficiency. This was a single-center randomized, double-blind crossover phase II clinical trial of SGS-742 versus placebo in patients...

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Veröffentlicht in:	Journal of child neurology 2021-11, Vol.36 (13-14), p.1189-1199
Hauptverfasser:	Schreiber, John M., Wiggs, Edythe, Cuento, Rose, Norato, Gina, Dustin, Irene H., Rolinski, Rachel, Austermuehle, Alison, Zhou, Xiangping, Inati, Sara K., Gibson, K. Michael, Pearl, Phillip L., Theodore, William H.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Amino Acid Metabolism, Inborn Errors Child Child, Preschool Cross-Over Studies Developmental Disabilities Double-Blind Method Female GABA Antagonists - therapeutic use Humans Male Organophosphorus Compounds - therapeutic use Succinate-Semialdehyde Dehydrogenase - deficiency Succinate-Semialdehyde Dehydrogenase - drug effects Treatment Outcome Young Adult
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