The Importance of Early Diagnosis in Wolman Disease
New Publication Expands Knowledge of Wolman Disease and Reports a Novel LIPA Variant A recent article published in Frontiers in Pediatrics reports on two Chinese infants diagnosed with Wolman Disease, the most severe infantile form of Lysosomal Acid Lipase...
Research and Hope for LAL-D
New Research Project Explores Innovative Strategies for Lysosomal Acid Lipase Deficiency (LAL-D) At LAL-D Patient Organization, we would like to congratulate Matthias Zadory on the successful defense of his doctoral thesis, “Engineering Non-Viral Gene...
New Findings from the International LAL-D Registry
New International LAL-D Registry Study Shows Sustained Improvements in Lipid Profiles with Sebelipase Alfa Treatment A new study published in Atherosclerosis provides important long-term evidence on the impact of sebelipase alfa enzyme replacement...
