Review developments in Pompe disease, expert opinion and discussion, and more in the following pages. Highlights and forthcoming expert content focus on the 2022 World Muscle Society Congress which was held in Halifax, Canada 11–15 October.
The aim of this study is to compare the effectiveness and safety of clinical outcomes between capsule surgery and conservative treatment in patients with TCS. A study hypothesis is that outcomes will be improved after capsule surgery.
The investigators hypothesize that by applying a validated algorithm to accomplish early mobilization in surgical intensive care unit (ICU) patients, these patients will achieve a higher level of mobility...
In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are...
Gender dysphoria describes the psychological distress caused by identifying with the sex opposite to the one assigned at birth. In recent years, much progress has been made in characterizing the needs of transgender persons...
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Drugs appearing in this section are approved by UK Medicines & Healthcare Products Regulatory Agency (MHRA), & the European Medicines Agency (EMA).