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NEUROLOGY 1981;31:288
© 1981 American Academy of Neurology

Double-blind, triple-crossover trial of low doses of oral physostigmine in inherited ataxias

R. A. Pieter Kark, M.D., Maria M. R. Budelli, M.D. and Robin Wachsner EM

From the Department of Neurology (Drs. Kark and Budelli), and the Department of Psychiatry (Dr. Wachsner), Reed Neurological, and Jerry Lewis Neuromuscular (Drs. Kark and Budelli), and Mental Retardation Research Centers (Drs. Kark, Budelli and Wachsner), UCLA School of Medicine, Los Angeles, CA 90024.

The signs of ataxia in patients with inherited ataxias have been reported to improve after single injections or single oral doses of physostigmine. To study this effect, 21 patients with various inherited ataxias underwent a randomized, double-blind, triple-crossover trial of physostigmine and inert placebo taken orally in low doses for four sequential 3-month periods. Overall, the drug was more effective than the placebo (p <0.051. Thirteen patients had consistent, statistically significant responses to physostigmine; 8 did not (x2=46.9, p <0.0001). Only three patients improved on placebo. Responses did not correlate with age, sex, or severity of ataxia. Some aspects of ataxia improved in some patients, other aspects in other patients. Preliminary accounts of this work were previously reported.1-2

Address correspondence and reprint requests to Dr. Kark, Reed Neurological Research Center, UCLA School of Medicine, Los Angeles, CA 90024.

This study was supported in part by grants from the National Institutes of Health, HD-06576, HD-04612, and RR-865; by grant13 from the Muscular Dystrophy Association Drug Task Force; a Muscular Dystrophy Association Research Fellowship to Dr. Budelli; by a grant from the Louis B. Mayer Foundation; and by donations from Mrs. Marjorie L. Johnson and the Friedreich's Ataxia Group in America.

Accepted for publication May 21, 1980.







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