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These sobering facts require the pursuit of: (1) Newborn screening to diagnose and treat infants in the first week or two of life

A truncated 67-aa IGF-1 analog lacking the N-terminal Gly-Pro-Glu tripeptide, with higher intrinsic IGF-1R affinity and moderately reduced IGFBP binding

4, 37 rare disease treatments had been approved by the FDA in 2023, according to stats provided to BioSpace by the National Organization for Rare Disorders