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Phthalazinol, Thrombocytopenia, and Amyotrophic Lateral Sclerosis
W. King Engel, MD;
Benjamin Rix Brooks, MD
Arch Neurol. 1980;37(5):320.
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| Since this article does not have an abstract, we have provided the first 150 words of the full text PDF and any section headings. |
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Side effects of a drug sometimes lead to new uses. Phthalazinol is an inhibitor of cyclic adenosine monophosphate (cAMP) phosphodiesterase (and cyclic guanine monophosphate (cGMP) phosphodiesterase to a much lesser extent), with no serious side effects.1-3 We have used phthalazinol, 30 to 75 mg/kg/day, in patients during a period of four years, for as long as 33/4 years in one patient, attempting to treat amyotrophic lateral sclerosis.1 Phthalazinol raised to normal the low CSF levels of cAMP, but did not raise the low levels of cGMP.3.4 Phthalazinol did not benefit the disease in 20 patients.3 One 69-year-old man with only bulbar involvement, which had progressed for 30 months before treatment, has had no further progression during 47 months of phthalazinol therapy, 35 mg/kg/day. Whether arrest was related to the phthalazinol or a spontaneous plateauing of his disease is not yet known.3 Transient side effects in a
. . . [Full Text PDF of this Article]
Author Affiliations
From the Neuromuscular Diseases Section, National Institute of Neurological and Communicative Disorders and Stroke, Bethesda, Md.
Footnotes
Accepted for publication July 30, 1979.
Reprint requests to Neuromuscular Diseases Section, National Institute of Neurological and Communicative Disorders and Stroke, Bldg 10, Room 10D20, Bethesda, MD 20205 (Dr Engel).
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