About Morquio Syndrome
Morquio syndrome (MPS IV) is characterized by severe skeletal dysplasia with normal intelligence, distinguishing it from other MPS disorders. MPS IVA (GALNS deficiency) is the most severe form, causing short stature, odontoid hypoplasia with cervical instability, joint laxity, and corneal clouding. MPS IVB is milder. Elosulfase alfa (Vimizim) is approved as ERT for MPS IVA, improving endurance and respiratory function.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Morquio Syndrome trials.
Cervical spine MRI/CT is required before enrollment in any Morquio trial due to high risk of cervical myelopathy
Elosulfase alfa (Vimizim) ERT is standard for MPS IVA — document prior ERT history and infusion-related reaction history
Six-minute walk test (6MWT) and pulmonary function tests are standard eligibility and outcome measures
MPS IVA versus MPS IVB must be confirmed by GALNS enzyme activity and/or GLB1 genotyping
Patient Resources
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