Fear Related to Access Issues in Acid Sphingomyelinase Deficiency (ASMD)

• With the recent FDA approval of olipudase alfa for acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, it is important to understand the
access burdens experienced by patients and families in order to plan for support services that allow families to obtain coverage of this new treatment option
• The patient support organization, the National Niemann-Pick Disease Foundation (NNPDF) recently conducted a study which examined health insurance
literacy and access burdens in Niemann-Pick disease
• This poster discusses the access burdens reported by respondents representing 24 patients who had been diagnosed with ASMD