Recent years have seen tremendous progress towards therapy of many previously incurable
neuromuscular diseases. This new context has acted as a driving force for the development …

Lying at the intersection between neurobiology and epigenetics, Rett syndrome (RTT) has
garnered intense interest in recent years, not only from a broad range of academic scientists …

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …

Background Duchenne muscular dystrophy (DMD) is a hereditary neuromuscular disease
leading to progressive muscle wasting. Since there is a need for MRI variables that serve as …

Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy. In the
present study, when human induced pluripotent stem cells (hiPSCs) were differentiated into …

Recent advances in bioengineering have enabled cell culture systems that more closely
mimic the native cellular environment. Here, we demonstrated that human induced …

Introduction The system of clustered regularly interspaced short palindromic repeats
(CRISPR) and CRISPR-associated proteins (cas) is a new technology that allows easier …

In Duchenne muscular dystrophy (DMD) little has been reported on the association between
clinical outcome measures and patient health-related quality of life (HRQOL) tools. Our study …

Introduction: Duchenne muscular dystrophy (DMD) is an X-linked handicapping disease due
to the loss of an essential muscle protein dystrophin. Dystrophin-null animals have been …

A ready source of autologous myogenic cells is of vital importance for drug screening and
functional genetic studies in Duchenne muscular dystrophy (DMD), a rare disease caused …