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Exploring New Treatment Advances for Acid Sphingomyelinase Deficiency in Adults
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... |
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Cleveland Clinic Hospital Medicine
Cleveland Clinic Hospital Medicine features a comprehensive review of the most common and relevant conditions seen in the inpatient setting. A globally renowned faculty addresses the application of new evidence, interprets current guidelines and provides management strategies which can improve the quality of medical care. All physicians, nurses and physician assistants treating hospitalized patients will benefit from this thoughtful assessment of best practices in this critical area of medici... |
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