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Inborn errors of metabolism elicit unique challenges for therapy development
As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
来自MSN2 个月
ACMG sets new lifelong guidelines for managing phenylalanine hydroxylase deficiency
Revolutionizing care for phenylalanine hydroxylase (PAH) deficiency, ACMG's updated guidelines highlight evidence-based strategies to enhance neurocognitive health and protect maternal and fetal ...