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The result is a catch-22: patients must persist long enough to demonstrate clinical benefit, yet the financial burden often prevents that persistence, undermining the very outcomes needed to justify reimbursement
doi:10.1016/j.jhepr.2024.101095 Harlow TR, PharmDa, Peters
Both CBS OPT and L2HGDH OPT are about 7KDa larger than the endogenous ones
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Storage guide for lyophilized powder, reconstituted vials, fridge timing, freezing, and discard signs
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