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Real-World Data Assessment of Safety of Home-Based and Hospital/Outpatient-Based laronidase Enzyme Replacement Therapy for Mucopolysaccharidosis I

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source: Molecular genetics and metabolism reports

year: 2021

authors: Wilson A,Colzani RM,Flores AL


Enzyme replacement therapy (ERT) is available for several lysosomal storage disorders to address the underlying lysosomal enzyme deficiencies that lead to the accumulation of undegraded substrates in multiple organ systems. A typical ERT regimen is a 2–6 h weekly intravenous infusion. ERT administered in a home setting has been reported by patients and caregivers to be both less stressful and more convenient than hospital or clinic-based administration. Laronidase (recombinant human α-L-iduronidase; Aldurazyme®) is approved in over 75 countries for the treatment of the non-neurological manifestations of mucopolysaccharidosis I (MPS I).

Patients with MPS I may opt to receive their ongoing laronidase treatment at home, but to date there has been no formal comparison of safety of home-based versus hospital/outpatient-based infusions in the United States (US). To address this gap, real-world data were retrospectively analyzed from two medical and pharmacy insurance claims databases to assess safety of home-based and hospital/outpatient-based laronidase ERT.

organization: Sanofi Genzyme, USA.

DOI: 10.1016/j.ymgmr.2021.100736

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