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Investigators from Eli Lilly designed escalation schedules to balance maximum receptor saturation with patient compliance [1, 3]
Refrigerating the vial after room-temperature exposure doesn't restore lost potency
These trials are still ongoing
doi:10.1001/jama.2021.1831 Lundgren JR, Janus C, Jensen SBK, et al
Although the use of specialized amino acid formulations that contain minimal to no valine, isoleucine, methionine, and threonine have become widely implemented, these types of diets can lead to iatrogenic amino acid deficiencies and poor growth outcomes in many methylmalonic acidemia patients
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