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STATEMENT OF NEED
Acid sphingomyelinase deficiency (ASMD) is a rare, hereditary lysosomal storage disease with multisystemic manifestations, including splenomegaly, hepatomegaly, interstitial lung disease, dyslipidemia, bone marrow and lymph node involvement, and sometimes neurological and peripheral nerve involvement. While the most severe form of ASMD, infantile neurovisceral ASMD, results in death in early childhood, chronic forms of ASMD have more prolonged survival but are associated w...
i3 Health
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