scholarly journals Real-world data assessment of safety of home-based and hospital/outpatient-based laronidase enzyme replacement therapy for mucopolysaccharidosis I

2021 ◽  
pp. 100736
Author(s):  
Amanda Wilson ◽  
Raffaella M. Colzani ◽  
Ana Lorena Flores
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Real World ◽  
Real World Data ◽  
World Data ◽  
Mucopolysaccharidosis I ◽  
Hospital Outpatient ◽  
Enzyme Replacement ◽  
Home Based ◽  
Data Assessment

Enzyme-Replacement Therapy in Mucopolysaccharidosis I

2001 ◽  
Vol 344 (3) ◽  
pp. 182-188 ◽  
Author(s):  
Emil D. Kakkis ◽  
Joseph Muenzer ◽  
George E. Tiller ◽  
Lewis Waber ◽  
John Belmont ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Mucopolysaccharidosis I ◽  
Enzyme Replacement

Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings

2013 ◽  
Vol 109 (3) ◽  
pp. 315-316 ◽  
Author(s):  
Sarah Laraway ◽  
Catherine Breen ◽  
Jean Mercer ◽  
Simon Jones ◽  
James E. Wraith
Keyword(s):  
Natural History ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Mucopolysaccharidosis I ◽  
Enzyme Replacement ◽  
Early Use ◽  
History Of

Home-based service for enzyme replacement therapy in lysosomal storage disorders: patient reported outcomes

2018 ◽  
Vol 6 (4) ◽  
pp. 669
Author(s):  
Paolo Tirelli ◽  
Fiorina Giona ◽  
Maja Di Rocco ◽  
Elena Cassinerio ◽  
Antonio Pisani ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Lysosomal Storage Diseases ◽  
Lysosomal Storage ◽  
Nursing Service ◽  
Enzyme Replacement ◽  
Home Based ◽  
Patient Reported

Background:  Lysosomal storage diseases (LSDs) are a heterogeneous group of rare chronic genetic conditions. The standard-of-care treatment for LSDs is hospital-based infusion of enzyme replacement therapy (ERT), however, over time this can be stressful and inconvenient. The Italian TuTor program, established in 2011 by Sanofi Genzyme, is a professional nursing service providing home-based ERT to patients with LSDs.Objectives:  The current questionnaire-based study was conducted to investigate the level of patient satisfaction with theTuTor program and to shed light on disease perception.Methods:  Patients were enrolled in the TuTor program from 2011 onwards. The first 100 patients enrolled were interviewed at baseline with follow-up interviews conducted at 6, 12 and 18 months.Results: Overall, 52 patients were female; 46 had Gaucher’s disease, 46 had Fabry disease and 8 had mucopolysaccharidosis type 1. Patients took on average >2 hours to receive hospital-based ERT, plus time associated with the infusion; 2 out of 3 patients needed a caregiver to travel to the hospital. After receiving home-based ERT for 6 months, 37% of patients considered their quality of life ‘greatly improved’ (60% at 18 months). Overall, 99% to 100% of patients rated the home-based nursing service as ‘positive’ or ‘very positive’ and reported that they would recommend the service to other patients with their condition.Conclusions: For patients with LSDs eligible for ERT, a disease-specific home-based nursing service increased their perception of quality of life over a hospital-based service and was advantageous in terms of their time and expenditure.

scholarly journals Long-term outcomes of enzyme replacement therapy for Taiwanese patients with Mucopolysaccharidosis I

2019 ◽  
Vol 60 (5) ◽  
pp. 577-578 ◽  
Author(s):  
Hsiang-Yu Lin ◽  
Chung-Lin Lee ◽  
Chih-Kuang Chuang ◽  
Shuan-Pei Lin
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Long Term Outcomes ◽  
Mucopolysaccharidosis I ◽  
Enzyme Replacement
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