MPS (RaDiCo Cohort) (RaDiCo-MPS)

Anyone of any age, male or female, can take part in this study. The most important thing is that you have a confirmed diagnosis of one of the specific MPS conditions that the researchers are looking at.

To be considered for the study, doctors need to have already found problems with specific enzymes in your body. They also need to have found unusual levels of certain sugar molecules (called GAG) in your urine, or have identified specific genetic changes (mutations) in your DNA that confirm you have MPS.

If you are under 18 or someone who needs help making decisions, a parent or guardian will need to give their permission for you to be included. If a patient is no longer alive, their parent or guardian can still give permission for their medical records to be included in the study.

As this is an 'observational' study, it’s different from studies that test new medicines. You won't be given any new treatments or asked to change your current medical care. Instead, taking part mainly involves allowing the research team to collect information from your existing medical records. This might include details about your diagnosis, symptoms, and how your condition has progressed over time.

If you are still receiving care, the research team might also collect information from your routine clinic visits. You might have some extra assessments during these regular appointments, but you won't need to make special trips just for the study. There are no study medications to take, and no specific follow-up appointments outside of your usual care. The total duration of your participation could depend on how long your medical records are available or how long you continue to be seen in clinic.