Phenylketonuria
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Sign up freeTop drugs studied for this disease
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FDA approval · 2013-12-19FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for KUVAN (sapropterin dihydrochloride) submitted by BioMarin Pharmaceutical, resulting in updated l…
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CLINICAL_TRIALS trial_result · 2013-12-05Clinical Trial Update: Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
A clinical trial evaluating the behavioral effects of Kuvan (sapropterin dihydrochloride) in children with mild phenylketonuria has been ter…
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FDA approval · 2013-11-22FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for KUVAN (sapropterin dihydrochloride) submitted by BioMarin, resulting in an updated labeling for …
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CLINICAL_TRIALS trial_result · 2013-09-10Clinical Trial Update: Follow-up of Adult Phenylketonuria (PKU) Patients
This clinical trial update reports the completion of follow-up in adult phenylketonuria (PKU) patients. The snippet does not provide detaile…
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CLINICAL_TRIALS trial_result · 2013-08-01Clinical Trial Update: Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period [Phase 4]
Phase 4 trial update examining response to Kuvan (sapropterin dihydrochloride) in subjects with phenylketonuria over a 4-week testing period…
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FDA approval · 2013-01-23FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for KUVAN (sapropterin dihydrochloride) from BioMarin Pharmaceutical, signaling an update to its…
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CLINICAL_TRIALS research · 2013-01-18Clinical Trial Update: Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
This Clinical Trial Update reports that a study evaluating Kuvan for individuals with phenylketonuria and maladaptive behaviors has complete…
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CLINICAL_TRIALS trial_result · 2012-11-16Clinical Trial Update: Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 [Phase 3]
A Phase 3 trial of Phenoptin in phenylketonuria patients who participated in PKU-004 or PKU-006 has completed and results have been posted.
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CLINICAL_TRIALS trial_result · 2009-08-20Clinical Trial Update: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels [Phase 3]
This update reports the completion of a Phase 3 multicenter open-label extension trial of Phenoptin in individuals with phenylketonuria and …
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CLINICAL_TRIALS trial_result · 2009-07-09Clinical Trial Update: Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet [Phase 3]
Phase 3 study evaluating Phenoptin to increase phenylalanine tolerance in children with phenylketonuria on a restricted diet has completed, …
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CLINICAL_TRIALS trial_result · 2009-04-16Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels [Phase 3]
Phase 3 trial evaluating safety and efficacy of Phenoptin in individuals with phenylketonuria completed; results have been posted.
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FDA approval · 2008-06-20FDA Approved: KUVAN — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for Kuvan (sapropterin dihydrochloride) by BioMarin Pharmaceutical Inc.
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CLINICAL_TRIALS approval · 2008-04-15Clinical Trial Update: Sapropterin Expanded Access Program
Sapropterin (Kuvan) has been approved for marketing for phenylketonuria under an expanded access program.
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FDA approval · 2007-12-13FDA Approved: KUVAN — Original Application (BioMarin Pharmaceutical Inc.)
FDA approved Kuvan (sapropterin dihydrochloride) from BioMarin as an original application for the treatment of phenylketonuria, to improve t…
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CLINICAL_TRIALS trial_result · 2007-09-20Clinical Trial Update: Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety [Phase 2, Phase 3]
A terminated Phase 2/3 trial evaluated long-term tetrahydrobiopterin treatment in children with phenylketonuria to assess phenylalanine tole…
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CLINICAL_TRIALS trial_result · 2007-04-09Clinical Trial Update: Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria [Phase 2]
Phase 2 trial evaluating the response to and safety of an 8-day course of Phenoptin for phenylketonuria has completed.