| Abstract: | The use of guanidine hydrochloride is reported in the treatment of 4 cases of Werdnig-Hoffmann disease. Improvement was achieved in two cases. Amelioration was initially observed in the third patient, but a relapse occurred during temporary discontinuation of therapy and the disease progressed, albeit at a slower rate, after resumption of therapy. The final case was a terminal one in which, however, slight temporary improvement occurred in response to therapy. A maximum dosage of 0.4 g (based on the assumption of an average body weight of 15 kg in these children) was reached by gradual increments. Side effects like shortlasting vomiting were observed only in case 1. The pharmacological action of guanidine hydrochloride is discussed. According to Otsuka and Endon the mechanism of action may be based on an increase in the end-plate potential amplitude. However, an augmented supply of spinal transmitters may also play a role in the mediation to the pharmacological action. |
