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Description
DBdouble blind, SBsingle-blinded, PCplacebo-controlled, Hbhemoglobin, IVintravenous, kgkilograms, mgmilligrams, ggrams, molmicromole, Lliters, Lp (a)lipoprotein a, LDLlow-density lipoprotein, HDLhigh-density lipoprotein, TGtriglycerides, TCtotal cholesterol, HDhemodialysis, VLDLvery-low-density lipoprotein

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While a number of promising therapies for SMA are emerging, defining therapeutic windows and developing sensitive and relevant biomarkers are critical to facilitate potential success in clinical trials
