How Does the Registry Work?

Patients

What is a Registry? About Lysosomal Acid Lipase Deficiency (LAL-D)

Why Participate? How Do I Participate Registry Sponsor Patient Organizations

Data Entry and Collection

Informed Consent

To be able to participate in the registry, your doctor will have to explain the purpose of the registry and your role in participating. After communicating with your doctor, and fully understanding the registry, you will provide informed consent prior to data entry authorization.

The patient may at any time opt out of participating in the registry.

Data Sharing

Your doctor will enter your data through a secure site into the Registry database.

Collected Information

Your information will be given a unique ID number, and only your physician will know which data belongs to you. The registry does not collect information that identifies you, such as your name.

Collected Information

Once submitted, your data is reviewed for accuracy. This involves certain electronic checks (to prevent, for example, a 6 month old infant having data entered that indicates he is pregnant) as well as manual checks by a Registry’s data management team member to ensure the data entered is accurate, consistent, and plausible. If the data entered is outside possible ranges, the patient’s doctor may be asked to confirm the data.

Information Available for Research

Once the data has been reviewed, it will be available in the Registry for research into LAL-D. Information will not be used to identify individuals in research, but will be used as grouped information. In addition to the analyses of grouped information, your doctor may be able to generate individual reports to help monitor your disease and optimize your management plan. Your doctor can share these reports with you.