Methods and compositions for the treatment of metabolic disorders
AssigneeBiomarin Pharmaceutical Inc.
InventorsDaniel Oppenheimer, Emil Kakkis, Fredric Price, Alejandro Dorenbaum
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Abstract
The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
Claims93
A method for treating classic severe phenylketonuria (PKU) in a subject comprising administering to said subject a protein-restricted diet in combination with a composition comprising tetrahydrobiopterin (BH4) or a precursor or derivative thereof, wherein the combined administration of the protein-restricted diet and BH4 is effective to lower the phenylalanine concentration in the plasma of said subject as compared to said concentration in the absence of said combined administration.
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A method for the treating a pregnant female having hyperphenylalaninemia (HPA) comprising administering to said subject a protein-restricted diet in combination with a composition comprising tetrahydrobiopterin (BH4) or a precursor or derivative thereof, wherein the combined administration of the protein-restricted diet and BH4 is effective to lower the phenylalanine concentration in the plasma of said subject as compared to said concentration in the absence of said combined administration.
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A method of treating a patient having above normal concentration of plasma phenylalanine comprising administering to said patient a stabilized BH4 composition in an amount effective to produce a decrease in the plasma phenylalanine concentration of said patient.
A method of treating a patient having phenylketonuria, comprising administering to said patient a stabilized BH4 composition in an amount effective to produce a decrease in the plasma phenylalanine concentration of said patient wherein said patient has been diagnosed as unresponsive to a BH4 loading test.
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A method of treating an infant having phenylketonuria, comprising administering a stabilized BH4 composition to said patient in an amount effective to produce a decrease in the plasma phenylalanine concentration of said infant wherein said infant is between 0 and 3 years of age and said infant has a plasma phenylalanine concentration of between about 360 μM to about 4800 μM.
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A composition comprising a stabilized, crystallize form of BH4 that is stable at room temperature for more than 8 hours and a pharmaceutically acceptable carrier, diluent or excipient.
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Dependencies are inferred from the claim text.