Orphan designation: (S)-N-(2-(2-Cyclopropylpyridin-4-yl)-4,5,6,7-tetrahydrobenzo[d]oxazol-4-yl)-1-methyl-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide Treatment of KCNT1-related epilepsy, 20/05/2026 Positive

Submitted by Anonymous (not verified) on 8 July 2026 - 9:24

Orphan designation: (S)-N-(2-(2-Cyclopropylpyridin-4-yl)-4,5,6,7-tetrahydrobenzo[d]oxazol-4-yl)-1-methyl-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide Treatment of KCNT1-related epilepsy, 20/05/2026 Positive

Orphan designation: antisense oligonucleotide against USH2A pre-mRNA Treatment of inherited retinal disease due to dysfunction in the USH2A gene, 20/05/2026 Positive

Submitted by Anonymous (not verified) on 8 July 2026 - 9:24

Orphan designation: antisense oligonucleotide against USH2A pre-mRNA Treatment of inherited retinal disease due to dysfunction in the USH2A gene, 20/05/2026 Positive

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