Why Participate

Patients

What is a Registry? About Lysosomal Acid Lipase Deficiency (LAL-D) How Does the Registry Work?

Why Participate?

How Do I Participate Registry Sponsor Patient Organizations

Registries can contribute to the understanding in any disease area, and they are particularly important for rare disorders such as LAL-D, where they provide a means to gather evidence and increase the collection of information. Participation of patients in the registry is critical to each registry’s success. For registries to be most useful, they need to include as many patients as possible.

The LAL-D Registry is open to all people with LAL-D, regardless of whether they are receiving treatment or not. The LAL-D Registry began in 2013 to help better understand LAL-D and its management.

LAL-D is passed from affected parents to their children, therefore if you are diagnosed with LAL-D, there is a chance that other members of your family may have it too. Siblings of a child with LAL-D have a 1 in 4 chance of also being affected. Additionally LAL-D is a rare disease, and registries can help better understand what is happening in patients with LAL-D.

With the help of patients with LAL-D, together we can help the lives of those living with LAL-D.

* Patient image is hypothetical

By collecting information of people with LAL-D in a central database, research can be conducted on larger numbers than would otherwise be possible, so we may learn things that will enable better care for patients in the future.

Research using the Registry’s data will be overseen by the Scientific Board of Advisors, a group of physicians with expertise in LAL-D. The advisors regularly review the registry information, and have presented medical-scientific registry results at medical conferences.