Use of Ambroxol as Therapy for Gaucher Disease.
JAMA network open(2023)
摘要
In this case series of ambroxol repurposing among patients with GD, long-term treatment with ambroxol was safe and associated with patient improvement. Improvements in hematologic parameters, visceral volumes, and plasma biomarkers were larger among patients with relatively mild symptoms of GD and patients who received initial treatment at younger ages.
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关键词
ambroxol,disease
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