• Doctors discuss SMA, including cause, inheritance, diagnosis, and progression
  • Animations detail the science of SMA, including RNA splicing
  • Researchers explain the science behind emerging therapies for SMA
  • SMA patients and families talk about diagnosis and living with SMA

Learn About SMA is a resource for spinal muscular atrophy (SMA) patients, families and researchers. The site includes stories of living with SMA and recent advances in the understanding and potential treatment of SMA.

Learn About SMA is divided into five sections that can be browsed in a non-linear fashion, with video interviews, animations, and narrative.

What is SMA? includes interviews with doctors and patients, plus an animation explaining the cause, inheritance and diagnosis of SMA. SMA Science provides an introduction to the genes and mechanisms involved with SMA, including 2-D and 3-D animations and interviews with Nobel Laureates. In SMA Therapies doctors discuss current and potential treatments for SMA and a father describes the daily routine of physical therapies for his daughter, who has SMA. Antisense Therapy for SMA includes videos and animations to explain antisense therapy for SMA. In Living with SMA four SMA families describe daily routines, disease progression, children's understanding of SMA, and grieving.

SMA News: Team uses antisense technology that exploits gene splicing mechanism to kill cancer cells