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22 This highlights the importance of continuous pharmacist involvement to assess tolerability, determine when dose adjustments or therapy changes are warranted, and guide patients through individualized management strategies
ERT is also potentially limited by reaccumulation of Gb3 in podocytes after dose adjustment during the follow-up period and formation of neutralizing antidrug antibodies after infusion, which reduce the efficacy of ERT by increasing cellular Gb3 deposition and results in harmful clinical outcomes that include progressive loss of renal function 13,14
Instead, it offers patients two ways to access the drugs: By affiliate links into a direct-to-consumer portal from the manufacturer, or by printing out a coupon that can be redeemed at a pharmacy
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