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Lorscheider J, Buzzard K, Jokubaitis V, Spelman T, Havrdova E, Horakova D, et al

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European Study Group on Growth Hormone Treatment in Children with Nephropathic Cystinosis (2001) Long-term treatment with growth hormone in short children with nephropathic cystinosis

Indeed, the clinical literature exhibits heterogeneity in design, dosing, duration, and outcome measures, frequent reliance on parent reported scales without uniform blinding, and underpowered samples that hinder strong inference and guideline development (Doherty et al., 2022