Uses: Primary systemic carnitine deficiency Acute and chronic treatment of inborn errors of metabolism resulting in secondary carnitine deficiency Prevention and treatment of carnitine deficiency in end stage renal disease patients undergoing dialysis (IV form only) Renal Dose Adjustments Safety and efficacy have not been evaluated in patients with renal insufficiency
But, its way too early to tell at this point. Arnold says other states may have the flexibility to consider mandating ACA insurers to cover the drugs
Mignini, F., Streccioni, V., Tomassoni, D., Traini, E., and Amenta, F
Prakoso D, De Blasio MJ, Tate M, Kiriazis H, Donner DG, Qian H, Nash D, Deo M, Weeks KL, Parry LJ, Gregorevic P, McMullen JR, Ritchie RH (2020) Gene therapy targeting cardiac phosphoinositide 3-kinase (p110alpha) attenuates cardiac remodeling in type 2 diabetes
The STEP 1 trial measured outcomes at week 68, roughly sixteen months