How ASMD Can Impact Quality of Life and Average Life Expectancy​

ASMD can lead to morbidity and early mortality¹

^aData 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 untreated patients with ASMD compared to the general population³

ASMD can impact quality of life^1,4

ASMD is associated with a high burden of disease that may limit physical, mental, and psychosocial well-being. A patient’s experience may include^1,4:

• 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

^bBased on data from a natural history study of 10 patients with ASMD type A, during which all 10 patients died.^5
cBased 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.⁶

References: 1. McGovern MM et al. Orphanet J Rare Dis. 2017;12(1):41. 2. McGovern MM et al. et al. Orphanet J Rare Dis. 2021;16(1):212. 3. Pulikottil-Jacob R et al. Mol Genet Metab Rep. 2023;38:101040. 4. Cox GF et al. JIMD Rep. 2018;41:119-129. 5. McGovern MM et al. Neurology. 2006;66(2):228-232. 6. Cassiman D et al. Mol Genet Metab. 2016;118(3):200-213.