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Drug Pipeline
RCT
Sepiapterin reduces blood phenylalanine more than sapropterin in PKU patients
Sepiapterin beats sapropterin for lowering blood levels
This Phase 3, randomized, crossover trial in 82 children and adults with phenylketonuria compared sepiapterin to sapropterin.
New research shows a specific vitamin treatment works much better than current options for a rare genetic condition.
May 1, 2026
Pediatrics
Cohort
Excessive maternal zinc supplementation may exacerbate fetal copper deficiency in Menkes disease
A Common Pregnancy Supplement May Worsen a Rare Baby Disease
This review evaluates the interaction between excessive maternal zinc supplementation and Menkes disease in fetuses with ATP7A dysfunction.
Doctors are asking if a popular over-the-counter supplement could accidentally harm babies with a hidden genetic condition.
Frontiers
Apr 13, 2026
Diabetes & Endocrinology
RCT
Walking program improves liver enzymes and BMI in children with PKU and NAFLD
Walking program shows improvement in liver health markers for children with PKU and fatty liver
An 8-week RCT in 40 overweight/obese children with PKU and NAFLD found that a free walking program significantly improved BMI, ALT, GGT, ALP…
An 8-week walking program improved liver health markers and BMI in children with PKU and fatty liver disease.
Apr 3, 2026