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Ambroxol Repurposing Improves Biochemical Markers in Gaucher Disease

Significant improvements seen in hematologic parameters, visceral volumes, and plasma biomarkers in patients with Gaucher disease, suggesting its potential as an alternative therapy, particularly for those who cannot access enzyme replacement therapy.
Xia Zhan Shanghai Jiao Tong University School Of Medicine Department Of Pediatric Endocrinology Shanghai Institute For Pediatric Research Pediatric Endocrinology Xinhua Hospital

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