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Clinical trials for testing new drugs are currently ongoing, such as a new GCS inhibitor with limited off target activity (NCT06114329), as well as testing for new types of treatments involving stem cell transplant with CD34 positive cells transfected with lentivirus to produce -galactosidase A (NCT02800070) [30], or new drugs to combine with traditional treatments, like dapaglifozin for patients with cardiac and renal involvement (NCT05710367)

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References Won Son, J