0960-8966/$ see front matter 2013 Elsevier B.V. All rights re http://dx.doi.org/10.1016/j.nmd.2013.07.013 We read with interest the recent debate on steroids, growth and motor function decline in Duchenne muscular dystrophy (DMD) [1–3]. The efficacy and safety of growth hormone treatment in 39 ambulant DMD boys with glucocorticoid-induced growth failure was recently evaluated [3]. The authors o...

Progress in understanding the role of dystrophin raises promising hopes for a treatment for Duchenne muscular dystrophy. In addition, great improvements have been made in the ability to diagnose this disease using simple molecular methods.

To cite: Behera V, Behera MK, Chauhan R, et al. BMJ Case Rep Published online: [please include Day Month Year] doi:10.1136/bcr-2014205296 DESCRIPTION A 15-year-old boy presented with progressive proximal weakness of the lower limbs starting at 4 years of age followed by involvement of the upper limbs. He is the product of a consanguineous marriage; he had a family history of similar disease in ...

Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction, is a promising novel therapy for genetic diseases of muscle and nerve-the prototypical example being Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common childhood genetic disease, affecting one in 3500 newborn boys, causing progressive muscle ...