Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study

Harvey L. Levy, Andrzej Milanowski, Anupam Chakrapani, +9

The Lancet

Sapropterin DihydrochlorideRenal FunctionChronic Kidney DiseasePharmacologyPhase Iii +4

Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

Friedrich K. Trefz, Barbara K. Burton, Nicola Longo, +16

The Journal of Pediatrics

Sapropterin DihydrochlorideMedicineInherited Metabolic DiseaseClinical NutritionPharmacology +4

The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6<i>R</i>‐tetrahydrobiopterin): a phase II, multicentre, open‐label, screening study

Barbara K. Burton, Dorothy K. Grange, Andrzej Milanowski, +8

Journal of Inherited Metabolic Disease

Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria

Phillip Lee, Eileen P. Treacy, Eric Crombez, +11

American Journal of Medical Genetics Part A

Sapropterin DihydrochlorideMetabolic DisorderPharmacotherapyMetabolic SyndromeTranslational Medicine +14

Recovery of Malassezia pachydermatis from eight infants in a neonatal intensive care nursery

Mark LaRocco, Alex Dorenbaum, Ann Robinson, +1

The Pediatric Infectious Disease Journal

Malassezia FurfurAntibioticsPediatrics15-Month Retrospective SurveySepsis +7

Concentrations of protease inhibitors in cord blood after in utero exposure

Mark Mirochnick, Alex Dorenbaum, Diane Holland, +7

The Pediatric Infectious Disease Journal

Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: Results of a phase 3b study

Barbara K. Burton, Maria Nowacka, Julia B. Hennermann, +8

Molecular Genetics and Metabolism

Extended TreatmentSapropterin DihydrochlorideUrologyMedicinePharmacology +3

Pharmacokinetics of Sapropterin in Patients with Phenylketonuria

François Feillet, L. Clarke, Concetta Meli, +8

Clinical Pharmacokinetics

Renal FunctionMedicineChronic Kidney DiseasePharmacotherapyNephrology +2

Relative bioavailability of sapropterin from intact and dissolved sapropterin dihydrochloride tablets and the effects of food: A randomized, open-label, crossover study in healthy adults

Donald G. Musson, William G. Kramer, Erik D. Foehr, +4

Clinical Therapeutics

NutritionDietary ExposureMedicineSapropterin Dihydrochloride TabletsToxicology +4

The Impact of Race/Ethnicity on Mother-to-Child HIV Transmission in the United States in Pediatric AIDS Clinical Trials Group Protocol 316

Coleen K. Cunningham, Rajalakshmi Balasubramanian, Isaac Delke, +8

JAIDS Journal of Acquired Immune Deficiency Syndromes

EthnicityPuerto RicoReproductive HealthHealth DisparitiesMother-to-child Hiv Transmission +22