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Disease Burden

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ASMD can lead to morbidity and early mortality1

Disease progression and complications may result in premature death2,3

ASMD type B patients have ~30% reduced survival probability compared to the US general population

*Data extrapolated from a Kaplan-Meier curve generated in a 11-year natural history study that evaluated morbidity and mortality in 59 patients with ASMD type B. At entry, 30 patients were in the pediatric age group (<18 years of age) and 29 patients were adults (≥18 years of age). There were 9 deaths during the follow-up period. Reduction in survival probability is absolute, not relative. US General Population as of 2017.2

Reduction in average life expectancy of patients with ASMD compared to the general population3-5

Reduction in life expectancy of patients with ASMD types A, A/B, and B compared to the US general population

ASMD can impact quality of life1,6

ASMD is associated with a high burden of disease that may limit physical, mental, and psychosocial well-being. A patient’s experience may include1,6:

  • Frequent hospitalizations, medications for symptomatic care, medical procedures, and increased need for medical devices
  • Reduced physical activity and difficulty navigating obligations at school, work, and in personal relationships
  • Feelings of social isolation and rejection

†Based on data from a natural history study of 10 patients with ASMD type A, during which all 10 patients died.5

‡Based on a natural history study of 85 patients with ASMD, 27 patients were identified with ASMD type A/B and 58 patients were identified with ASMD type B. Of the 27 patients with ASMD type A/B, 26 patients died. Of the 58 patients with ASMD type B, 52 patients died.4

ASMD is a genetic disease
ASMD is a
genetic disease1
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References:
  1. McGovern MM, Avetisyan R, Sanson BJ, Lidove O. Orphanet J Rare Dis. 2017;12(1):41.
  2. McGovern MM Wasserstein MP, Bembi B, et al. Orphanet J Rare Dis. 2021;16(212):1-14.
  3. Arias E, Xu J. Centers for Disease Control and Prevention website. 2017; Vol 28, No. 7. https://www.cdc.gov/nchs/products/index.htm.
  4. Cassiman D, Packman S, Bembi B, et al. Mol Genet Metab. 2016;118(3):200-213.
  5. McGovern MM, Aron A, Brodie SE, Desnick RJ, Wasserstein MP. Neurology. 2006;66(2):228-232.
  6. Cox GF, Clarke LA, Giugliani R. JIMD Rep. 2018;41:119-129.