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: Ataluren Delays Loss of Ambulation and Decline in Pulmonary Function in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

Researchers

Presenter

  • Craig Mcnald

Principal Investigators

  • Francesco Muntoni

  • Mark Rance

  • Joel Jiang

  • Allan Kristensen

  • Vinay Penematsa

  • Francesco Bibbiani

  • Elizabeth Goodwin

  • Heather Gordish-Dressman

  • Lauren Morgenroth

  • Richard Able

  • Panayiota Trifillis

  • Mar Tulinius

Medical Centers

  • UC Davis Dept. of PM&R

  • UCL-Institute of Child Health

  • T3D Therapeutics Inc.

  • T3D Therapeutics Inc.

  • Children's National Hospital

  • TRiNDS

  • Goteborg University,

Locations

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Musculoskeletal

  • Genetic Disorder

Disease

  • Duchenne muscular dystrophy

Biomarkers

  • N/A

Drug/Treatment

  • Deflazacort

  • Ataluren

Outcome

  • N/A

Study Design

  • N/A

Phase

  • III

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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