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Data from the International LAL-D Registry reported long term outcomes in 29 patients (12 male, 17 female) with rapidly progressive infantile-onset LAL-D treated with sebelipase alfa (47)

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The Faseb Journal, 35(1)

Another described resolution of chronic H

Thus, we propose QMP engineering as a strategy to ensure a long plasma half-life and enhanced therapeutic efficacy of albumin fusion proteins despite C-terminal cleavage