hrp0092rfc3.6 | Multi-system Endocrine Disorders | ESPE2019

Prevalence of Endocrine Complications in Duchenne Muscular Dystrophy

Kotnik Primož , Jazbinšek Sončka , Golli Tanja , Osredkar Damjan

Background: Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene and results in a progressive muscular damage and degeneration. Endocrine complications result from decreased energy expenditure, immobility and glucocorticoid (GC) treatment. Due to the multidisciplinary management and emerging genetic and molecular therapies longer survival is expected and there is an increasing emphasis on the quality of life in DMD. Aim of the study ...