Long-Term Outcomes of Disease Modifying Therapies in Gaucher Disease

ConclusionsAwareness about the disease and the efficacy of the therapies amongst pediatricians will help in early diagnosis and better outcomes. The available therapies have changed the outcome of the patients and improved the quality of life in patients with Gaucher disease. The data of Indian patients is important at this juncture when under Rare Disease Policy, government funding has become available for ERT for Gaucher disease patients in India.

http://link.springer.com/10.1007/s12098-023-04986-y

Source: Indian Journal of Pediatrics - February 5, 2024 Category: Pediatrics Source Type: research