Morquio A Registry Study (MARS)


Morquio A

Full Study Name

Morquio A Registry Study (MARS)



Lead Site

Birmingham Children’s Hospital

Chief Investigator

Saikat Santra

Local Principal Investigator

Elisabeth Jameson

Study Status


Main Aims

To characterize and describe the MPS IVA population as a whole, including the variability between patients, progression of the disease over time and natural history of MPS IVA.

Inclusion Criteria
  • Diagnosed with MPS IVA as confirmed by either GALNS enzymatic test or by a diagnostic molecular test.
  • Willing and able to provide written, signed informed consent, or in the case of patients age < 18 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the Registry has been explained, and prior to any Registry-related procedures.
  • Willing to undergo entry assessments to establish baseline data or permit Investigator to enter assessment data recorded prior to Registry entry if available in the patient’s medical records.

Entry assessments may include:
demographics, medical history, urinary keratan sulfate level, urinary protein level, immunogenicity testing, vital signs, physical examination, and height and weight.

Exclusion Criteria
  • Patients currently participating in a BMN 110 (elosulfase alfa) clinical trial
Open Sites

Birmingham, Salford, Manchester, Great Ormond Street, UCLH, Belfast, Dublin, Royal Free, UCLH