Research and Clinical Trials

The Lysosomal Acid Lipase Deficiency Registry

The LAL-D Registry is a place where your doctor can enter information about LAL-D patients' medical condition but with no identifying information. This is essential, especially since LAL-D is rare because it helps researchers to learn more about the characteristics of LAL-D to increase scientific knowledge and lead to new discoveries. Please ask your doctor if you would like to be enrolled in the LAL-D registry so that your signs and symptoms or your child's can be including in the body of knowledge on LAL-D. 

To learn more about the LAL-D Registry please visit ClinicalTrials.Gov LAL-D Registry

LAL-D Registry

Clinical Trials

Synageva BioPharma Corporation was bought by Alexion Pharmaceuticals, which continues to study sebelipase alfa under the commercial name Kanuma, a potential enzyme replacement therapy for LAL D. More information about clinical trials can be found at www.clinicaltrials.gov (search term “LAL Deficiency)”.

https://clinicaltrials.gov/ct2/results?term=lal+deficiency&Search=Search

On September 1, 2015, Alexion announced that the European Commission approved Kanuma for the treatment of patients of all ages with LAL-D. The FDA granted Breakthrough Therapy designation for Kanuma for LAL Deficiency presenting in infants and accepted the Kanuma BLA for Priority Review. In addition, a New Drug Application for Kanuma has been submitted to Japan’s Ministry of Health, Labour and Welfare.

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