A NOVEL TREATMENT REGIMEN INVOLVING SAPROPTERIN DIHYDROCHLORIDE

A NOVEL TREATMENT REGIMEN INVOLVING SAPROPTERIN DIHYDROCHLORIDE

The invention relates to an improved tetrahydrobiopterin formulation for treating patients affected by hyperphenylalaninemia. More specifically, the formulation of the invention permits a prolonged residence of tetrahydrobiopterin in the plasma, and/or a higher concentration of sapropterin in the pl...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: GELBHART, Bareket, CANNATA, Fabio, PAIN, Gilles
Format: Patent
Sprache:eng
Schlagworte:
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Beschreibung
Zusammenfassung:The invention relates to an improved tetrahydrobiopterin formulation for treating patients affected by hyperphenylalaninemia. More specifically, the formulation of the invention permits a prolonged residence of tetrahydrobiopterin in the plasma, and/or a higher concentration of sapropterin in the plasma, enabling notably the treatment of HPA patients that would usually not be classified as responders to tetrahydrobiopterin loading test, and therefore left without any pharmacological treatment opportunity, but only with a strictly controlled protein diet.