Which of the following is granted orphan drug status for the treatment of Dravet’s syndrome?
Correct Answer: Stiripentol
Description: Stiripentol
Stiripentol increases GABA transmission. It is granted orphan drug status for the treatment of Dravet’s syndrome (Severe myoclonic epilepsy in infants).
Icatibant is a bradykinin B2 receptor antagonist. It is used for acute attacks of hereditary angioedema in adults with C1-esterase inhibitor deficiency.
Pitolisant (a/k/a Tiprolisant) is a inverse agonist of H3 receptor. It is under trial for schizophrenia and Parkinson’s disease.
Tafamidis stabilizes the correctly folded tetrameric form of TTR. It is used in familial amyloid polyneuropathy.
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