Guanidine hydrochloride in infantile and juvenile spinal muscular atrophy. A double blind controlled study

A double-blind controlled study was conducted to assess the effect of guanidine hydrochloride on infantile and juvenile spinal muscular atrophy. Twenty patients (twelve treated with guanidine and eight given placebo) were followed up for up to one year, and muscular strength was evaluated by manual muscle testing. The most frequent side effects of the drug were paraesthesiae and anaemia. In all our five patients affected by infantile spinal muscular atrophy with prolonged survival, guanidine treatment resulted in a clinical stabilization. No effect was observed in the Kugelberg-Welander group.