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Thirty Years Beyond Discovery—Clinical Trials in Succinic Semialdehyde Dehydrogenase Deficiency, a Disorder of GABA Metabolism

This review summarizes a presentation made at the retirement Symposium of Prof. dr. Cornelis Jakobs in November of 2011, highlighting the progress toward clinical trials in succinic semialdehyde dehydrogenase (SSADH) deficiency, a disorder first recognized in 1981. Active and potential clinical inte...

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Bibliografische gegevens
Gepubliceerd in:J Inherit Metab Dis
Hoofdauteurs: Vogel, Kara R., Pearl, Phillip L., Theodore, William H., McCarter, Robert C., Jakobs, Cornelis, Gibson, K. Michael
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2012
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4349389/
https://ncbi.nlm.nih.gov/pubmed/22739941
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-012-9499-5
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