Phenylketonuria
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Sign up freeTop drugs studied for this disease
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FDA approval · 2016-06-13FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental application for Kuvan (sapropterin dihydrochloride), confirming FDA authorization of changes includ…
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CLINICAL_TRIALS trial_result · 2016-05-23Clinical Trial Update: Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis [NA]
A completed clinical trial from the University of Glasgow examining how a phenylketonuria-type diet affects appetite, appetite hormones, and…
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CLINICAL_TRIALS trial_result · 2016-05-12Clinical Trial Update: Kuvan® in Phenylketonuria Patients Less Than 4 Years Old [Phase 3]
Phase 3 trial update for Kuvan in phenylketonuria patients under 4 years old; study completed with results posted in 2016.
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CLINICAL_TRIALS trial_result · 2016-04-20Clinical Trial Update: A Pilot Study on Diurnal Variation [NA]
This clinical trial update summarizes results from a terminated pilot study investigating diurnal variation in Phenylketonuria (PKU).
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CLINICAL_TRIALS trial_result · 2016-02-17Clinical Trial Update: Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
A completed clinical trial update evaluating neurocognitive outcomes in individuals with mild hyperphenylalaninemia/phenylketonuria; the sni…
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CLINICAL_TRIALS trial_result · 2016-02-12Clinical Trial Update: Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria [Phase 2]
A Phase 2 trial evaluating sapropterin for cognitive abilities in young adults with phenylketonuria was terminated with results posted in 20…
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CLINICAL_TRIALS trial_result · 2016-02-08Clinical Trial Update: Phenylketonuria and Hyperphenylalaninemia Nutrition Study
This entry reports the completion of a nutrition-focused clinical trial studying phenylketonuria and hyperphenylalaninemia. The provided sni…
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CLINICAL_TRIALS trial_result · 2016-02-01Clinical Trial Update: Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients [Phase 3]
Phase 3 study evaluating the safety and potential neuropsychiatric benefits of sapropterin dihydrochloride in phenylketonuria patients; the …
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CLINICAL_TRIALS trial_result · 2015-12-11Clinical Trial Update: Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
A completed clinical trial examining the biological variation of phenylalanine levels in patients with hyperphenylalaninemia/phenylketonuria…
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CLINICAL_TRIALS trial_result · 2015-12-11Clinical Trial Update: Testing of Four Home Phenylalanine Monitoring Prototype Devices
A completed clinical study evaluated four prototype devices for at-home phenylalanine monitoring in people with phenylketonuria.
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CLINICAL_TRIALS trial_result · 2015-12-11Clinical Trial Update: The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU) [NA]
A completed clinical trial investigated whether supplemental docosahexaenoic acid (DHA) affects neurocognitive outcomes in teen and adult wo…
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FDA approval · 2015-10-28FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin's supplemental application for KUVAN (sapropterin dihydrochloride).
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CLINICAL_TRIALS trial_result · 2015-07-07Clinical Trial Update: Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy [NA]
This clinical trial update reports bone mineral density changes in adults with hyperphenylalaninemia treated with Kuvan. The study (NCT01541…
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CLINICAL_TRIALS trial_result · 2015-06-19Clinical Trial Update: The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study [NA]
This clinical trial update reports results from a pilot study evaluating brain structure, neurological features, and neuropsychological func…
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CLINICAL_TRIALS trial_result · 2015-06-12Clinical Trial Update: Bone Mineral Density in Adults With Hyperphenylalaninemia
This clinical trial update assesses bone mineral density in adults with hyperphenylalaninemia (phenylketonuria). The update summarizes the b…
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CLINICAL_TRIALS trial_result · 2015-05-12Clinical Trial Update: Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy [NA]
This clinical trial update reports FDG-PET imaging findings in patients with phenylketonuria before and after treatment with Kuvan (sapropte…
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CLINICAL_TRIALS withdrawal · 2014-12-17Clinical Trial Update: Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria [NA]
A clinical trial investigating neuroimaging and neurocognitive responses to sapropterin dihydrochloride in phenylketonuria has been withdraw…
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CLINICAL_TRIALS trial_result · 2014-10-16Clinical Trial Update: Moderate Intensity Exercise and Phenylketonuria [NA]
A clinical trial investigating moderate-intensity exercise in phenylketonuria has been terminated.
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CLINICAL_TRIALS trial_result · 2014-08-28Clinical Trial Update: Phenylketonuria, Oxidative Stress, and BH4 [Phase 2]
A Phase 2 trial evaluating tetrahydrobiopterin (BH4) in phenylketonuria was terminated, with results posted in 2014.
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CLINICAL_TRIALS trial_result · 2014-08-20Clinical Trial Update: Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
A completed Emory University clinical trial studied nutritional and neurotransmitter changes in people with phenylketonuria treated with tet…
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CLINICAL_TRIALS trial_result · 2014-08-20Clinical Trial Update: The Early History of Universal Screening for Metabolic Disorders
This completed clinical trial update from the University of Miami provides historical context on the early universal screening programs for …
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CLINICAL_TRIALS trial_result · 2014-07-28Clinical Trial Update: Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria [Phase 3]
A Phase 3 open-label study evaluating the response and safety of Kuvan (sapropterin dihydrochloride) in subjects with Phenylketonuria has co…
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FDA approval · 2014-04-23FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin's supplemental application for Kuvan (sapropterin dihydrochloride). The approval updates the drug's labeling and/or ex…
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CLINICAL_TRIALS withdrawal · 2014-04-23Clinical Trial Update: The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU
Clinical trial update indicating the study evaluating sapropterin dihydrochloride supplementation for in vivo redox status in classical phen…
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FDA approval · 2013-12-19FDA Approved: KUVAN — Original Application (BioMarin Pharmaceutical Inc.)
The FDA approved Kuvan (sapropterin dihydrochloride) from BioMarin as the original approval for treatment of phenylketonuria to lower blood …