September 10, 2025NutrientsOpen Access

Sapropterin Dihydrochloride Responsiveness in Phenylketonuria: A Case Series Exploring Gaps in Comprehensive Patient Monitoring

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Case series analyzed sapropterin effects on dietary tolerance in PKU, suggesting adherence to best practices is vital.

All patients showed significant reductions in blood phenylalanine levels, averaging 71.43%, indicating effective sapropterin treatment.
With a criterion of a ≥30% reduction in phenylalanine levels, most patients achieved better dietary tolerance, showing impressive outcomes.
Analysis of 23 past cases treated with sapropterin over several years revealed gaps in adherence to international guidelines for monitoring.
While effective, the treatment protocol deviated from guidelines, underscoring the need for improved long-term neuropsychological assessments.

Bianco et al. (2025) studied this question.

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