Fig. 1

Study design. VA velmanase alfa. ^aIf applicable in accordance with routine clinical practice, unscheduled follow-up visits can be performed at, but not limited to, 3 months after VA treatment start, or whenever deemed appropriate according to clinician judgment for patients who start VA treatment within 1 year prior to registry inclusion. ^bIf a patient is transferred to another continuing care site during the 15-year observational period, the new treating site will be formally involved in the registry following a request of authorization submitted to the relevant ethics committee and regulatory authority to complete the 15-year observational period. ^cPatients can start VA treatment at any time during the course of their participation in the registry; if applicable in accordance with routine clinical practice, the baseline visit should be repeated before administration of VA and unscheduled visits recommended as reported in the schema above