Table 1 General statements

Statement 1

ERT with elosulfase alfa should be available for all MPS IVA patients independent of age and genetic variant

100

Statement 2

Treatment with elosulfase alfa should be initiated as early as possible

100

Statement 3

Baseline assessments as well as regular follow-up assessments should be performed as described in the guidelines:

 1. 6MWT or another appropriate test

 2. uKS levels

 3. pulmonary function (FVC, FEV1)

 4. cardiac function (LVEF)

 5. PROs

  QoL: MPS-HAQ Caregivers Domain, EQ-5D-5L

  Pain: Brief Pain Inventory (BPI)

  Depression: Beck Depression Inventory (BDI)

100