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Long-term effects of eliglustat on skeletal manifestations in clinical trials of patients with Gaucher disease type 1

Timothy M. Cox, Joel Charrow, Elena Lukina, Pramod K. Mistry, Meredith C. Foster, Michel Peterschmitt

2022Genetics in Medicine20 citationsDOIOpen Access PDF

Topics & Concepts

MedicineEnzyme replacement therapyDiseaseClinical trialSurgeryInternal medicineLysosomal Storage Disorders ResearchTrypanosoma species research and implicationsGlycogen Storage Diseases and Myoclonus