An 18-month report on the safety and efficacy of rapid intravenous velaglucerase alfa infusions in naïve patients with Gaucher disease
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DOI: 10.1016/j.ymgme.2021.11.038
Abstract: The introduction of enzyme replacement therapy (ERT) has revolutionized the management of patients with Gaucher disease (GD). To improve the patients' quality of life, we have studied and published the safety and efficacy of a… read more here.
Keywords: month; safety efficacy; velaglucerase alfa;
The budget impact of enzyme replacement therapy in type 1 Gaucher disease in the United States
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DOI: 10.1080/13696998.2022.2082200
Abstract: Abstract Aim Gaucher disease (GD) is a rare autosomal recessive condition. Type 1 GD (GD1) is the most prevalent form of GD in Western countries; enzyme replacement therapy (ERT) is a treatment option for patients… read more here.
Keywords: alfa; velaglucerase alfa; budget impact;