Phase 2 Start~month

Phase 2 Start: A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Effi

Drug
gne
Indication
GNE Myopathy

Other upcoming events for RARE

PDUFA DatePDUFA Date: dtx401 aav gene therapy
Phase 3 ReadoutPhase 3 Topline: An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Eva
Phase 3 ReadoutPhase 3 Topline: An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphona
Phase 3 ReadoutPhase 3 Topline: A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared With
Phase 3 ReadoutPhase 3 Topline: A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving th

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    Phase 2 Start: A Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Effi — RARE Phase 2 Start | BioSniper