Becker MD Natural History Study

• Research type

  Research Study

• Full title

  PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

• IRAS ID

  170233

• Contact name

  Michela Guglieri

• Contact email

  michela.guglieri@ncl.ac.uk

• Sponsor organisation

  Cooperative International Neuromuscular Research Group (CINRG)

• Duration of Study in the UK

  4 years, 0 months, 0 days

• Research summary

  Becker Muscular Dystrophy(BMD) is a known milder form of a genetically inherited muscle wasting condition affecting males. The gene responsible is the dystrophine gene, which in a more severe form is responsible for Duchene Muscular dystrophy. This is a life limiting condition affecting the walking muscle , leading to early loss of walking and requiring a wheelchair.

  80 participants will take part in this world-wide study into the natural history of Becker Muscular Dystrophy. The study involves and 4 visits over 36 months of genetically confirmed BMD in order to compare their clinical phenotypes and health related outcomes. The data will inform a larger prospective study on the natural history of the disease.

• REC name

  Yorkshire & The Humber - Bradford Leeds Research Ethics Committee

• REC reference

  15/YH/0059

• Date of REC Opinion

  13 Mar 2015

• REC opinion

  Further Information Favourable Opinion