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News about "rare disease research"
Ionis Completes Enrollment in Phase 3 REVEAL Study for Angelman Syndrome
Ionis has completed enrollment in the pivotal Phase 3 REVEAL study of obudanersen for Angelman syndrome, with topline clinical trial results expected in the second half of 2027.
Rare Disease Research | 06/07/2026 | By Akanki | 160
Alltrna Receives Approval to Begin First-in-Human Trial of tRNA Therapeutic AP003
Alltrna initiates first-in-human trial of engineered tRNA therapy AP003 in Australia, marking a breakthrough in genetic disease treatment.
Rare Disease Research | 01/04/2026 | By News Bureau | 281
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