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08/08/2019
Dynacure Receives Orphan Drug Designation from the US FDA for DYN101, an Antisense Medicine to Treat Rare Disease ‘Centronuclear Myopathies’
Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Centronuclear Myopathies (CNM). Dynacure expects to initiate its first in human study, a Phase 1 / 2 study ‘Unite-CNM’, in the second half of 2019. DYN101 is being developed in collaboration with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery. [...]

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