Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial
Sapropterin dihydrochloride, a synthetic formulation of BH4, the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients ≥4 years with BH4-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®)...
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| Main Authors: | , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
9 March 2017
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| In: |
Orphanet journal of rare diseases
Year: 2017, Volume: 12, Pages: 1-11 |
| ISSN: | 1750-1172 |
| DOI: | 10.1186/s13023-017-0600-x |
| Online Access: | Verlag, kostenfrei, Volltext: http://dx.doi.org/10.1186/s13023-017-0600-x Verlag, kostenfrei, Volltext: https://doi.org/10.1186/s13023-017-0600-x 
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| Author Notes: | Ania C. Muntau, Alberto Burlina, François Eyskens, Peter Freisinger, Corinne De Laet, Vincenzo Leuzzi, Frank Rutsch, H. Serap Sivri, Suresh Vijay, Milva Orquidea Bal, Gwendolyn Gramer, Renata Pazdírková, Maureen Cleary, Amelie S. Lotz-Havla, Alain Munafo, Diane R. Mould, Flavie Moreau-Stucker and Daniela Rogoff |
| Summary: | Sapropterin dihydrochloride, a synthetic formulation of BH4, the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients ≥4 years with BH4-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) trial was to assess the efficacy (improvement in daily phenylalanine tolerance, neuromotor development and growth parameters), safety and pharmacokinetics of sapropterin dihydrochloride in children <4 years. |
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| Item Description: | Gesehen am 28.08.2018 |
| Physical Description: | Online Resource |
| ISSN: | 1750-1172 |
| DOI: | 10.1186/s13023-017-0600-x |