Table 1 Participant inclusion criteria

Parent/caregiver^a (or study participant if ≥ 18 years of age at the time of enrollment in our study)

&

Fluent in English (including non-native English speakers)

&

Able to give informed consent

&

With a confirmed diagnosis of ASMD

&

Used the experimental drug olipudase alfa for more than 12 months

&

Commenced treatment with olipudase alfa under the age of 18 years

&

Consented to participate in the study