What is a patient registry?

What is a patient registry?
  • Registries aim to...
    • ...facilitate patient recruitment for clinical trials - registered patients may be able to participate in trials more easily

    • ...accelerate research into new therapies for neuromuscular diseases (NMDs)

    • ...keep registered patients informed about research results, such as new treatments for NMDs

    • ...help specialists gain more knowledge about NMDs – how many people have it, where those people are and how the conditions progress over time

    • ...help achieve equal care for all patients with NMDs worldwide

    • ...provide a link to the community and provide patients with information directly relevant to their condition

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

When planning a clinical trial, it is very important that eligible patients can be found and contacted quickly. The best way for this to happen is through a database or "registry" that contains all the information that researchers will need. Patients' clinical and genetic details are collected and made easily available for the researchers.

Scientific advances over recent years have led to substantial changes in the treatment of many neuromuscular diseases. Several new therapeutic strategies which target specific genetic defects are being developed. Some of these treatments already have plans in place for large studies involving patients from more than one country.

Registries image

Any potential new treatment needs to be tested under strictly controlled circumstances to make sure, that not only are participants safe but, that any notable changes can be attributed to the treatment and not to external factors. This allows researchers to ensure that all potential new therapies are both safe and effective.

Due to the nature of rare diseases scientific approaches differ from common diseases. Finding enough patients that might be eligible to participate in trials for rare neuromuscular conditions can take years without a patient registry, delaying the testing of potential therapies.

12 Apr 2017