Abstract: Objective To observe the effectiveness and safety of mouse nerve growth factor in treatment of spinal muscular atrophy（SMA） in children. Methods A total of 18 children with SMA were treated by intramuscular injection with 9000AU mouse nerve growth factor from October 2006 to August 2007.Its safety was evaluated one month after treatment.Its therapeutic effect was quantitatively evaluated using the pediatric evaluation of disability inventory（PEDI） scale and electromyogram（EMG） three months after treatment. Results Of the 18 children who underwent the safety evaluation,7 underwent all the therapeutic effect evaluations of mouse nerve growth factor.No adverse effect was observed in all children.The clinical symptoms were improved at a certain extent.The muscle strength increased 0.5-fold.Improvements in SMA were found EMG and PEDI scale with no statistical significance. Conclusion Mouse nerve growth factor has certain therapeutic effects on SMA in children with no severe side effects.