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Fig. 4 | Orphanet Journal of Rare Diseases

Fig. 4

From: Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

Fig. 4

Perceived disease severity before and after the idursulfase-IT trial based on caregivers’ feedback. Only the total column reflects all interviews; age at study entry was not available for children outside the USA and Canada. a One participant whose child was in the age 2–6 years category was unable to provide a response to this question; one participant whose child’s age at study entry was unavailable was unable to provide a response to this question. b Two participants whose children were in the age 2–6 years category were unable to provide responses to this question; one participant whose child’s age at study entry was unavailable was unable to provide a response to this question. c Three participants, two of whom had children in the age 2–6 years category and one of whom had a child in the substudy, were unable to provide a response to this question; one participant whose child’s age at study entry was unavailable was unable to provide a response to this question. IT Intrathecal

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