DMD is an inheritable, X-chromosome linked, lethal childhood disease with a prevalence between 0.32 and 0.52 per 10,000 inhabitants. Worldwide, around 240,000 boys suffer from DMD. DMD is caused by mutations (often deletions) in the dystrophin gene that result in the disruption of the open reading frame leading to a loss of dystrophin protein expression.
Should you have any questions regarding BioMarin trials, or Duchenne Muscular Dystrophy please contact:
Paul Humphrey – Associate Director Patient Advocacy for the EU region: Paul Humphrey
Please also visit the BioMarin website for further details: http://biomarin.com
The following website also has excellent information on DMD, current research and trials: http://www.treat-nmd.eu/
