Skeletal health in Duchenne dystrophy: bone-size and subcranial dual-energy X-ray absorptiometry analyses.

INTRODUCTION: We performed subcranial and bone-size-adjusted whole body dual-energy X-ray absorptiometry (DXA) to evaluate skeletal health in Duchenne dystrophy (DMD). METHODS: Total body bone mineral density (TBBMD)-for-age, subcranial, and size-adjusted DXA analyses were performed on 22 DMD patients (5-17 years) and compared with 267 controls from a database. The skull contribution to total body bone mineral content (TBBMC) and corticosteroid effects were also examined. RESULTS: DMD boys had deficits in TBBMD-for-age (Z = -1.2), which increased with age. The skull's contribution to TBBMC decreased from 45% to 15% with growth. Z-scores for subcranial skeleton were significantly lower than TBBMC-for-area and TBBMD-for-age. CONCLUSIONS: Size-adjusted and subcranial analyses improve evaluation of whole body DXA. DMD boys have low BMD for size not commensurate with total body areal BMD-for-age. Bone fragility fractures in DMD may result from both decreased BMD and smaller bones. This information is vital to determine appropriate intervention. Muscle Nerve 49:512-519, 2014.

Outside in - inside out. Creating focus on the patient - a vaccine company perspective.

Involving patients in the development of medicines and vaccines should result in benefits to patients. The vaccine recipient is usually a healthy person. We describe the rationale and implementation of a vaccine company's initiative to encourage employees to identify with patients of the conditions prevented by the vaccines they help to produce. The Voice of the Patient ("VoP"), begun in 2014, is an educational programme directed at the 16,000 employees of a global vaccine company. It engages employees through an understanding that they are all "vaccine patients", and that they can make a difference by considering the impact of decisions made in their day to day work. The initiative includes presentations about vaccine-preventable diseases, global live webcasts with experts and patients, employee visits to healthcare facilities in developing countries, and the production of patient-focused sections in research publications. In a 2017 employee survey, 90% of respondents said they know how their daily work impacts patients and they demonstrate focus on patients. We believe this is preliminary evidence that, by supporting employee awareness of the impact of their individual roles, VoP could be a model for a type of initiative that will contribute to industry's continuing evolution towards more patient-centred healthcare.