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: Respiratory outcomes pre and post nusinersen in children with spinal muscular atrophy (SMA in a tertiary UK paediatric respiratory and neurology centre

Researchers

Presenter

  • Mihaela Diaconu

Principal Investigators

  • Chukwudumebi Duru

  • Kelechi Ugonna

  • Hemant Kulkarni

  • Min Ong

  • Kay White

Medical Centers

  • Sheffield Children's Hospital, Sheffield, United Kingdom

Locations

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Central Nervous System (CNS)

  • Genetic Disorder

Disease

  • Spinal muscular atrophy

Biomarkers

  • DNA Damage Inducible Transcript 3

Drug/Treatment

  • Nusinersen

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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