Global Registries

The importance of the Global Registries of Duchenne children 

There is an active global campaign with International scientific collaboration in the search for a cure for DMD. Current, ongoing research into new treatment therapies are showing real promise in either preventing or delaying the progress of the disease offering this generation of Duchenne children the hope of a different future and a life possibly decades longer. The International Registers of Duchenne patients are an essential component of this process. Accurate population based prevalence provides information to policy makers and health-care and education providers for the planning and provision of services to optimise quality of life for affected children and offer appropriate support for their families at all stages of the disease from diagnosis to death.

Crucially, the information held on these registers allows researchers to identify and contact the families of children whose profile fits the criteria for recruitment to a clinical trial. The aim of globally standardised care for Duchenne Muscular Dystrophy will contribute to an increased quality of life for the children and ensure that international variations in treatment and care will not make it difficult to interpret the results of the same clinical trials conducted in different countries.