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News and articles on spinal muscular atrophy

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

21 August 2015

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.

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SMA

Spinal muscular atrophy (SMA) is a genetically heterogeneous group of hereditary neurologic diseases and one of the types of motor neuron illness. Motor neurons are nervous cells of the spinal cord that set in motion the muscles necessary for executing motor functions of the human body.

The underlying mechanism of this disease is related to progressive degeneration of motor neurons located in anterior horns of the spinal cord, and in some cases – to motor nuclei of the brain stem. Every patient with SMA has a mutation in the survival motor neuron gene, producing a protein that is important for the normal functioning of motor neurons. Absence of this protein leads to atrophy of nervous cells, diminishing of their sizes and death resulting in muscle weakness.

Clinical symptoms of SMA are the following:

  • flaccid paresis and atrophy of striated muscles;
  • fasciculations (more often in adults with disease progression);
  • symmetric lesion of proximal muscles of extremities, less frequently distal amyotrophy, bulbar syndrome, and asymmetric muscle lesion;
  • problems with swallowing and suction in infants;
  • lesion of respiratory muscles that increases proneness to respiratory illnesses (pneumonia and other lung diseases);
  • spine deflection.

We have positive experience of treatment of acquired and genetic diseases affecting spinal motor neurons. Treatment with stem cell allows for attaining remission in 75% of cases, and for diminishing neurogenic dystrophy manifestations in SMA patients in 92% of cases.

Testimonials

Patient A.S., 9. Type 2 SMA, grade IV S-kyphoscoliosis

Patient M.S., 1. Spinal Muscular Atrophy, type I. Tetraparesis

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Fetal stem cells treatment results depend on: disease's severity, age of the patient, adherence for the medications and regime. Treatment results, presented on this site, are individual for each clinical case.

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