Acid Sphingomyelinase Deficiency (ASMD) is a rare lysosomal storage disease caused due to the deficiency of the enzyme acid sphingomyelinase. The deficiency results in the accumulation of sphingomyelin in cells and tissues and leads to a wide range of symptoms affecting multiple organs. The disease exists in two clinically distinct forms - the severe neonatal form (ASM-N) and the less severe but still life-threatening non-neurologic form (ASM-L). Most treatments for ASMD are aimed towards managing symptoms and improving quality of life rather than being curative. However, recent years have witnessed increased research activities to develop disease-modifying treatments.
The acid sphingomyelinase deficiency (ASMD) market is estimated to be valued at USD 134.6 Mn in 2024 and is expected to reach USD 300 Mn by 2031, growing at a compound annual growth rate (CAGR) of 12.1% from 2024 to 2031.
Advancements in the understanding of Acid Sphingomyelinase Deficiency (ASMD) Market disease pathophysiology and progress in gene and enzyme therapies are providing opportunities for development of transformative treatments for the condition.
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