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News and articles on spinal muscular atrophy

Judge allows stem cell cure for 2-year-old girl suffering spinal muscular atrophy 02 September 2012

Doctors are preparing an emergency one-off stem cell treatment for 2-year-old Venetian girl suffering a severe muscular disease after a judge revoked an order blocking the cure.

Understanding of Spinal Muscular Atrophy Improved With Use of Stem Cells 17 July 2012

Cedars-Sinai\'s Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy.

Possible Cause of Movement Defects in Spinal Muscular Atrophy Identified 15 April 2012

An abnormally low level of a protein in certain nerve cells is linked to movement problems that characterize the deadly childhood disorder spinal muscular atrophy, new research in animals suggests.

Hope for Spinal Muscular Atrophy Patients 01 December 2011

The experiments by Harvard Stem Cell Institute Therapeutic Screening Center resulted in the identification of approximately 150 molecules which raised the levels of protein called survival of motor neuron that drastically drops as SMA progresses.

All news

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.

Duration of the SMA treatment course at Cell Therapy Center EmCell, as a rule, totals 2 days.


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