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Coenzymes / Pteridines / Alcohols / Lactams / Tetrahydrobiopterin / Phenylketonuria / 6-Pyruvoyltetrahydropterin synthase deficiency / Phenylalanine hydroxylase / Pharmaceutical Benefits Scheme / Medicine / Health / Chemistry


Document Date: 2014-10-01 21:56:22

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Company

The PBAC / Merck Serono Pty Ltd / Merck Serono Australia Pty Ltd / Kao / /

Country

Japan / Canada / Australia / /

Currency

USD / /

Event

FDA Phase / Product Recall / Product Issues / M&A / /

IndustryTerm

sapropterin solution / tryptamine using gas / pterin-binding site / /

MedicalCondition

Inherited Metabolic Diseases / mild hyperphenylalaninaemia / Inherited Metabolic Disease / H Disorders / Atypical phenylketonuria / headache / dihydrobiopterin deficiency / tetrahydropterin synthase deficiency / such BH4 deficiency / mild mental retardation / Trial ID/First Author Protocol title/ Publication title BH4 deficiency / tetrahydrobiopterin synthase deficiency / dihydropteridine reductase deficiency / N Disorders / BH4 deficiency / tetrahydrobiopterin deficiency / phenylketonuria / peripheral deficiency / infections / Biopterin-dependent hyperphenylalaninaemia / chronic conditions / insomnia / incomplete 6pyruvoyl tetrahydropterin synthase deficiency / I deficiency / 6pyruvoyl-tetrahydropterin synthase deficiency / Atypical phenylketonuria treatment effectiveness / severe dystonia / Metabolic Diseases / nasopharyngitis / rare disease / Giovannini et al. Severe PTPS deficiency / PTPS deficiency / vomiting / pain / Proposed Therapy BH4 deficiency / Movement Disorders / allergic reactions / Synthase Deficiency / hyperphenylalaninaemia / PKU / Subtle brain dysfunction / deficiency / cough / Peripheral tetrahydrobiopterin deficiency / Disorders / upper respiratory tract infections / Ponzone et al. Dihydropteridine Reductase Deficiency / /

MedicalTreatment

alternative therapy / /

Organization

Pharmaceutical Benefits Advisory Committee / National Science Foundation / Australian Government / /

Person

Xue Za Zhi / /

Position

Physician / Trial ID/First Author / Governor / adult lawyer / Private / /

Product

Sapropterin / Kuvan / Reasons The PBAC / biopterin metabolism / BH4 / patients / S26-S30 / /

PublishedMedium

Pediatric Research / Archives of Neurology / /

Technology

gas chromatography / genotype / /

URL

http /

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