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

key information

source: Molecular genetics and metabolism reports

year: 2021

authors: Wilson A,Colzani RM,Flores AL

summary/abstract:

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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