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              Dynacure

              • Development Update

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              FRANCE France

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              Medicon Valley Inhalation Symposium

              Participation Not Confirmed

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              Dynacure Gets Rare Pediatric Disease Designation from U.S. FDA for DYN101 for Myotubular and Centronuclear Myopathies

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              Lead Product(s): Dynamin 101

              Therapeutic Area: Musculoskeletal Product Name: DYN101

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable July 30, 2020

              Details:

              U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2), for the treatment of Myotubular and Centronuclear Myopathies (CNM).

              Dynacure

              • Development Update

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              FRANCE France

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              Medicon Valley Inhalation Symposium

              Participation Not Confirmed

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              Dynacure Announces First Patient Dosed in Phase 1 / 2 'UNITE-CNM' Study of DYN101

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              Lead Product(s): DYN101

              Therapeutic Area: Musculoskeletal Product Name: Undisclosed

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 05, 2020

              Details:

              This clinical trial, based on Ionis' technology platform, is the first investigational study testing an antisense medicine to treat Centronuclear Myopathies.

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