Published: March 2025
Last updated: March 2025
PURPOSE: Qualitative accounts and studies employing disease-specific outcome measures have demonstrated that people with dystrophic epidermolysis bullosa (DEB) display impaired health-related quality of life (HRQoL) due to wound-related challenges. There...
AIMS: Asymptomatic severe aortic stenosis (aSAS) is associated with morbidity, with mortality increasing once symptoms develop. Benefits of early aortic valve replacement (AVR) over clinical surveillance (CS) with delayed AVR...
AIM: Late-onset Pompe disease (LOPD) is a rare lysosomal disease primarily impacting muscle strength andrespiratory function. LOPD has a substantial burden despite the availability of alglucosidase alfa (alg). Patients often...