Since the publication of the Duchenne muscular dystrophy (DMD) care considerations
in 2010, multidisciplinary care of this severe, progressive neuromuscular disease
has evolved. In conjunction with improved patient survival, a shift to more anticipatory
diagnostic and therapeutic strategies has occurred, with a renewed focus on patient
quality of life. In 2014, a steering committee of experts from a wide range of disciplines
was established to update the 2010 DMD care considerations, with the goal of improving
patient care. The new care considerations aim to address the needs of patients with
prolonged survival, to provide guidance on advances in assessments and interventions,
and to consider the implications of emerging genetic and molecular therapies for DMD.
The committee identified 11 topics to be included in the update, eight of which were
addressed in the original care considerations. The three new topics are primary care
and emergency management, endocrine management, and transitions of care across the
lifespan. In part 1 of this three-part update, we present care considerations for
diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and
adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.