This Plain Language Summary of publication article (PLSP) from Future Rare Diseases summarizes ASMD, a rare genetic disorder characterized by insufficient production of the enzyme acid sphingomyelinase, leading to the accumulation of sphingomyelin in organs like the lungs, liver, and spleen. The ASCEND study investigated the effectiveness of olipudase alfa, an enzyme replacement therapy, in adults with ASMD compared to a placebo.

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This PLSP is based on an article called ‘A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results’ and was published in Genetics in Medicine.

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