synthetic, single stranded, fully phosphorothioated 2’-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs)

synthetic, single stranded, fully phosphorothioated 2’-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs)
Trade Name
Orphan Indication Dravet Syndrome
USA Market Approval USA
USA Designation Date 2017-03-16 00:00:00
Sponsor OPKO Ireland Global Holdings Ltd.;10 Market Street, Suite 721;Camana Bay
Related Access Program
M. Scott Perry – Dravet Syndrome M. Scott Perry – Dravet Syndrome University of Colorado, Denver – Dravet Syndrome Children’s Hospital Medical Center, Cincinnati – Dravet Syndrome