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Journal: Advances in the design and discovery of novel small molecule drugs for the treatment of Dravet Syndrome. (Pubmed Central) - Oct 16, 2021
Experimental data and results from initial clinical studies have brought attention to several drugs with various mechanisms of action including: ataluren (a suppressant of premature stop codons; under clinical evaluation), EPX-100, EPX-200, fenfluramine (serotonin modulators), soticlestat (an 24-hydroxylase cholesterol enzyme inhibitor), SPN-817 (an inhibitor of acetylcholinesterase), verapamil (a voltage-dependent calcium channel inhibitor) and STK-001 (an antisense oligonucleotide). The latter is scheduled for clinical evaluation.
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