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Background: Children and adolescents with Pompe disease (PD) face chronic and progressive myopathy requiring time-intensive enzyme replacement therapy (ERT). Little is known about their perspectives on the disease and its treatment. This study…
Background Parents of neonates with life-threatening conditions and professionals, bear the burden of making complex decisions. Parents may not be fully involved in decision-making, and there is a paucity of evidence regarding the influence of social…