SMA is the leading genetic cause of death in infants, affecting approximately 1 in 10,000 infants. Types 1-4 have varying degrees of disability and expected mortality but all are degenerative and to date only supportive treatment options have existed. In the past 2-3 years research has brought forth several remarkable disease modifying treatment options that are now in the late stages of clinical development, nearing FDA approval, and potentially changing the outlook for this diagnosis entirely. This session will discuss the top 5-6 drug options in the later stages of research and clinical development referencing the 'CureSMA drug pipeline'. As these revolutionary treatments are anticipated to become more available through open and compassionate use trials and eventually FDA approval our patients and families will expect guidance from us, their providers. We will also discuss and engage the audience in the possible benefits and limitations of these treatments, how the development of these new treatments may impact our practice from practical discussions with patients and families, treatment approaches and providing resources.

Learning Objectives

  • Discuss the upcoming treatment options for SMA that are currently in the later stages of clinical development.
  • Discuss how these new treatments may impact this patient population in the future.
  • Provide a forum to discuss the opportunities as well as the limitations for treatment of current patients.