ArticleHealthcare (Basel, Switzerland)2023
Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic.
Article in Healthcare (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
3 citing papers in PubMed, 5 citations in OpenAlex.
- Artificial Intelligence, Connected Care, and Enabling Digital Health Technologies in Rare Diseases With a Focus on Lysosomal Storage Disorders: Scoping Review.Journal of medical Internet research · 2026Article
- Impact of the Covid19 pandemic on health-related quality of life in patients with Fabry disease - implications for future care of patients with rare diseases.Molecular genetics and metabolism reports · 2024Article
- Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials.Therapeutic advances in rare diseaseArticle
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivePatients with Lysosomal disorders (LSDs) are treated with regular infusions of enzyme replacement therapy (ERT). During the COVID-19 pandemic, home treatment was permitted. This study aimed at monitoring the patients' compliance with home therapy and its effects on physical, psychological, and relational issues. Moreover, we also tested the possible impact of home therapy on familial relationships and contacts with the referral hospital. MATERIALS AND
methodsThirteen patients with Pompe disease (N = 8) and MPS (N = 5) were tested through an online questionnaire designed to assess their level of appreciation and satisfaction with home therapy and their feelings about the referral centre and psychological support provided.
resultsMost of the patients (84%) stressed the positive impact of home therapy. All patients described a significant reduction in stressful conditions associated with the need to attend the hospital every week or two.
conclusionsHome ERT leads to a clear improvement in "daily life skills", as represented in our by sample by positive feelings, better emotional self-control, and an increased ability to understand the feelings of relatives. Our data underline the paramount positive effect home ERT has on both patients and their families.
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