Enzyme replacement therapy in Niemann-Pik Disease
050-9552 Enzyme Replacement Therapy in Niemann-Pick Disease
Compositions which increase the lysosomal sphingomyelinase activity in human subjects as a therapeutic for Niemann-Pick disease
Background:
Niemann-Pick disease types A and B (NPD-A/B) are severe, often fatal, hereditary lysosomal storage disorders characterized by a deficiency in the...
Published: 2/19/2026
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Inventor(s): Yusuf Hannun
Keywords(s):
Category(s): Campus > Stony Brook University
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