An interview with Natalia Rospara, president of the LAL-D Patients Association.
Enzyme replacement therapy allows LAL-D patients to function normally
Published June 4, 2024 08:00
A year ago, you founded the LAL-D (acid lysosomal lipase deficiency - editor's note) Patient Association. Please tell us how much this disorder affects patients' lives on a daily basis?
It is a multisystem, life-threatening disease with a number of complications. Untreated LAL-D can result in, for example: atherosclerosis, fibrosis, steatosis, liver failure or cirrhosis, as well as the death of the patient. The association is primarily made up of young people who have their whole lives ahead of them. I myself struggle with LAL-D, and thanks to the fact that I have treatment, I can be here and be the voice of all patients.
What is an unmet need for LAL-D patients?
It's the ability to get treatment and coordinated care.
What kind of treatment is it and how does it change the patient's perspective?
It is an enzyme replacement therapy that enables the patient to function normally in the first place, as well as facilitates daily life.
