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 the access burdens for patients diagnosed with Niemann-Pick disease

• This poster discusses the access burdens reported by respondents representing 24 patients diagnosed with ASMD