Amal AlQassmi
King Saud Medical City, Saudi Arabia
Posters & Accepted Abstracts: Health Care Curr Re
Introduction: Spinal muscular atrophy (SMA) is a major autosomal recessive neuromuscular disorder SMA causes motor neuron degeneration in the brain stem and spinal cord, leading to progressive muscle weakness and atrophy. The standard of care in SMA is mainly supportive and consists of nutritional and respiratory support, as needed, in addition to management of muscle weakness complications. Currently, there is Novel of therapy approved and present for SMA, showed their efficacy and progress of improvement. Based on one study done in Saudi addressed the cases recived therapy either Gene or Intrathecal Spinrazaâ?¦ We believe that, the entry of such novel, innovative, and high-cost treatments should be through an outcome-based agreement until the long-term follow-updata confirm real world effectiveness