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Stem cell therapy for three patients with spinal muscular atrophy: the outcomes

As there is no effective medical treatment for spinal muscular atrophy (SMA), stem cell treatment options draw more and more attention of the researchers and SMA community.

According to a recent study, three children with SMA type 1 underwent stem cell treatment, during which they received multiple infusions of mesenchymal stem cells, both intravenously and intrathecally. To evaluate efficacy of the treatment, pretreatment, treatment, and posttreatment physical function for two patients was measured by the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders and for all three - documented by video.

The results of the test were 3 before treatment and rose up to 10 and 16 during treatment for two patients, respectively. Seven and twelve months post-treatment, the test values decreased to 0 and 10.  

No adverse effects have been noted for at least 44 and 49 months from treatment onset for both patients, respectively.

Although the gains in test values were lost with the therapy discontinuation, this study may be a first step in proving mesenchymal stem cells injections as an effective and safe treatment for spinal muscular atrophy.