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  Cancer Studies

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Urea Cycle Disorder Studies

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.


Since 1987 Sodium Phenylbutyrate (generic name), 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 Sodium Phenylbutyrate.

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

Copies of clinical trials are available upon request for doctors and medical researchers.







DISCLAIMER:The information presented is intended for educational purposes for health professionals and practitioners, and is obtained from published research. It is not intended to be prescriptive nor to replace the care of a licensed health professional in the diagnosis and treatment of illness.
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