More About SMA

About SMA

  • SMA is the #1 genetic killer of young children.
  • Fewer than 1 in 35 people unknowingly carrying the gene responsible for SMA.
  • 1 in every 6,000 babies is born with SMA; SMA prevalence is comparable to ALS and Cystic Fibrosis.
  • SMA impacts the ability to walk, sit, stand, eat, breathe, and swallow. SMA does not impact the mind and children with SMA are bright and social.
  • There is currently no treatment and no cure for SMA, but there is HOPE!
  • The NIH has selected SMA as the disease closest to treatment of more than 600 disorders.
  • Leading U.S. researchers, including Nobel laureate James Watson, have stated that a viable treatment for SMA is possible within 5 years IF they are provided the proper resources.

About SMA Research

Although SMA is a relatively unknown common rare disease, scientific research and understanding of the disease is extremely mature and active. Dramatic breakthroughs have been made in the past fifteen years and, as a result of those breakthroughs, SMA has quickly gone from a poorly understood disease to being on the doorstep of a viable treatment. 		 

The SMA Treatment Acceleration Act will:

Provide Federal support for a national clinical trials network for SMA. Provide Federal support to enhance the SMA patient registry and for expanded research on the epidemiology of SMA. Establish an Interagency SMA Research Coordinating Committee to include federal agencies including NIH, SMA researchers, and SMA families, to coordinate government activities relating to SMA, develop a comprehensive strategy for improving and expanding SMA research, make recommendations to strengthen collaborative research across multiple institutes at NIH, and identify barriers to the development of drugs for treating SMA. Provide for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public. For more information on the SMA Treatment Acceleration Act visit GovTrack.us.

More About SMA

http://www.fsma.org/FSMACommunity/understandingsma/quickfacts/