Current Research Studies

Mucopolysaccharidosis Type IVA – Morquio A Registry Study (MARS)

Condition or Therapy:

Mucopolysaccharidosis Type IVA; Vimizim therapy

Category:

Genetics; Developmental Medicine 

What is the goal of this study?

The goal of this study is to:

  • Characterize and describe the Mucopolysaccharidosis (MPS) Type IVA population as a whole, including the heterogeneity, progression, and natural history of MPS IVA
  • Evaluate the long-term effectiveness and safety of Vimizim therapy
  • Help the medical community with the development of recommendations for monitoring MPS IVA patients and report on patient outcomes to optimize patient care

Who can join the study?

Any patient with a confirmed diagnosis of MPS IVA is eligible to join this study. 

What will happen if my child takes part in this study?

This study is a registry for MPS IVA patients. This registry collects relevant clinical data from participants medical records.

There are optional quality of life questionnaires that participants are asked to fill out.

There is also a small amount of blood that the study asks for to perform tests on. The amount of blood is equivalent to 2-3 tablespoons.

As always, participation is completely voluntary and participants can withdraw at any time, for any reason.

Who can I contact for more information?

Contact the study team by email or at 206-884-1264.


Study Location(s):

Seattle Children's Hospital main campus and Seattle Children's Bellevue Clinic and Surgery Center

Principal Investigator:

Dr. Angela Sun

Study posted: July 2, 2019