Sodium phenylbutyrate is an orphan drug, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl and by Swedish Orphan International (Sweden) as Ammonaps. It, along with special diets, has been used to treat urea cycle disorders. It also has received an orphan drug designation by the FDA for treatment of acute promyelocytic leukemia. It appears to have a number of potentially useful effects. Sodium phenylbutyrate was given an orphan drug designation by the FDA for use as an adjunct to surgery, radiation therapy, and chemotherapy for treatment of patients with primary or recurrent malignant glioma. Orphan drug designation has a lower burden of proof than that required for other FDA approved drugs. It has also been advocated for many years by the Burzynski clinic, more recently in combination with other drugs. Dr. Burzynski is conducting clincal trials with these combinations.
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