What is ASMD?
An inherited disease with multiorgan symptoms that can worsen over time
ASMD (acid sphingomyelinase deficiency) is historically known as Niemann-Pick disease types A, A/B, and B. This disease can be passed down through families, and has a wide array of symptoms that can impact your life in multiple ways. ASMD often affects the liver, lungs, spleen, and blood, as well as the digestive system.
There are 3 types of ASMD, which vary in symptom severity and appearance
||TYPE B (most common*)|
|First appearance of symptoms||Early infancy||Infancy to childhood||Infancy to adulthood|
|Organs involved||Affects many key organs in the body, including the brain||Affects many key organs in the body, including the brain||Affects many key organs
in the body, with little or
no impact to the brain
|Rate of symptom progression||Rapid||Varies in progression rate||Slow|
|Severity||Most severe||Varies in severity||Least severe|
*Based on patient population from a multicenter, historical cohort study (N=100).