Re: The budget impact of enzyme replacement therapy in type 1 gaucher disease in the United States

Editorial Material Endocrinology & Metabolism

Barth syndrome and the many fascinating aspects of cardiolipin

Frederic M. Vaz et al.

JOURNAL OF INHERITED METABOLIC DISEASE (2022)

Article Economics

The budget impact of enzyme replacement therapy in type 1 Gaucher disease in the United States

Sepehr Farahbakhshian et al.

Summary: This study estimated the budget impact of increasing the use of velaglucerase alfa compared to other enzyme replacement therapies for the treatment of Type 1 Gaucher disease in a hypothetical US healthcare plan. The results showed that increasing the use of velaglucerase alfa is cost-saving for US health plans.

JOURNAL OF MEDICAL ECONOMICS (2022)

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Article Hematology

Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results

Pramod K. Mistry et al.

Summary: Eliglustat, an oral substrate reduction therapy approved for Gaucher disease type 1, demonstrated significant clinical improvements in organ volumes and hematologic parameters over 4.5 years of treatment. Patients showed consistent improvements in disease manifestations and biomarkers, with a high level of tolerance to the medication. The results suggest that Eliglustat is an effective and well-tolerated treatment option for previously untreated patients with Gaucher disease type 1.

AMERICAN JOURNAL OF HEMATOLOGY (2021)

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Article Hematology