BACKGROUND: Patients with Pompe disease, a rare progressive neuromuscular disorder, receive a considerable amount of informal care. In this study, we examined the impact of providing informal care to patients with Pompe disease.
METHODS: Caregivers were administered various instruments, which measured the (impact of) informal care in the context of Pompe disease. Patients' quality of life and use of a wheelchair and respiratory support were used to investigate the impact of disease severity on the burden and well-being of caregivers.
