INBORN ERRORS OF UREA SYNTHESIS
(UREA CYCLE DISORDER)

triButyrate® was originally developed in the mid 1980's by triple crown america, inc., at the request of Johns Hopkins Hospital as a treatment for inborn errors of Urea Synthesis/Urea Cycle Disorder. For this indication triButyrate® (Sodium) Phenylbutyrate is today the Active Pharmaceutical Ingredient (API) of the registered drug to treat Urea Cycle Disorder in the United States of America.

Inborn errors of Urea Synthesis are rare and belong to the larger group of disorders of amino acid metabolism, which are genetically determined and caused by enzyme defects. The most well known disorder in the larger group is phenylketonuria (PKU), characterized by an accumulation of the amino acid phenylalanine, which frequently results in mental and psychomotor retardation, if not treated.

The disorders of inborn errors of Urea Synthesis are less known, but are life threatening conditions associated with hyperammonemia or high plasma glutamine levels, often resulting in coma and death in infants, if not treated immediately.

Non-Toxic
Since 1987 this compound, with generic name (Sodium) Phenylbutyrate, has been the treatment of choice for these diseases, recommended at a dosage of 450 to 500 mg/kg bodyweight per day. Approximately 1500 children worldwide are alive today and well because of triButyrate®.

EXTENSIVE TOXICOLOGY DATA derived from 15 years of continuous use and treatment at these dosage levels on these infants and children have proven (Sodium) Phenylbutyrate to be SAFE, EFFICACIOUS and with NO HARMFUL SIDE EFFECTS, even at the high dosage given over their individual lifetime. Toxicity starts appearing only at levels five to ten times larger than the commonly used dosages today for this disease, or at levels of 3g/kg bodyweight/day (approximately 200g/adult/day).

Natural Body Substance
Although triButyrate® is synthetically manufactured, once in the body it is quickly metabolized to a naturally occurring metabolite of phenylalanine. The fact that triButyrate® is converted to a natural body substance is considered to be one of the main reasons for its low toxicity.

All rights to the product and trademark triButyrate® are owned by
Fyrlklövern Scandinavia AB.



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