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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
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