Is there any new research being done on spinal muscular atrophy (SMA)?

Many scientists and clinicians are working hard to improve our understanding of this disease and improve the survival and quality of life of the SMA patient. Recent discoveries of the genes involved in SMA and the role(s) of these genes in maintaining the health of the motor neurons along with new developments in molecular medicine provide hope for better treatment strategies in this group of diseases. Research is now focused on strategies to enhance production of SMN protein through different approaches.

Last reviewed by a Cleveland Clinic medical professional on 11/24/2015.

References

  • Darras BT, Markowitz JA, Monani UR, De Vivo DC. Spinal muscular atrophies. In: Darras BT, Jones HR, et al, editors. Neuromuscular Disorders of Infancy, Childhood, and Adolescence. 2nd ed. Amsterdam: Academic Press, an imprint of Elsevier; 2014: Chap 8.
  • Amato AA, Russell JA. Spinal Muscular Atrophies. In: Amato AA, Russell JA. eds. Neuromuscular Disorders, 2e. New York, NY: McGraw-Hill; 2015.
  • Genetics Home Reference. Spinal muscular atrophy Accessed 11/24/2015.
  • National Human Genome Research Institute. Learning About Spinal Muscular Atrophy Accessed 11/24/2015.
  • Muscular Dystrophy Association (MDA). Spinal muscular atrophy Accessed 11/24/2015.

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