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What is a patient registry? 

A patient registry collects information about patients who are affected by a particular condition.

When a clinical trial is being planned, it is very important that patients suitable for that trial can be found and contacted quickly. The best way to insure that this happens is to make sure that patients’ details are all collected in a single database or “registry” that contains all the information that researchers will need, including each patient’s particular genetic defect and other key information about that condition.

A registry can also provide useful information on disease prevalence and the natural history of conditions.

Why do we need an international registry for FKRP-related conditions?

Patients affected by conditions caused by mutations in the Fukutin Related Protein (FKRP) gene are very rare.

Without a patient registry to gather patient details from all patients worldwide in one place, finding enough patients for a meaningful trial can take years, delaying the testing of potential therapies.

Patient numbers in different countries will be very low due to the rare nature of these conditions so it is more efficient to have one international registry that collects the information rather than having many national registries.