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City New York / Cambridge / / Company Nicholson AG / Genzyme Corporation / McGraw-Hill / Amicus Therapeutics / Ryder / Larkin AE / / Country United States / / / Facility National Center / Mount Sinai General Clinical Research Center / American College of Medical Genetics / Mount Sinai International Center / / IndustryTerm treatment of Niemann–Pick disease / imaging / therapy for Niemann–Pick disease / / MedicalCondition Pulmonary involvement / Pick disease Margaret M. McGovern / overall disease / Systemic lupus erythematosus / TB / Fauroux B. Lung disease / cholelithiasis / liver dysfunction / seizures / Alveolar lipoproteinosis / learning disability / liver failure / NP-B. Pulmonary / cirrhosis / thrombocytopenia / NP-B. Cardiac disease / endogenous lipid pneumonia / Wahn U. Asymptomatic pulmonary involvement / B Niemann-Pick disease / Odievre M. Fatal liver failure / Pulmonary / acute promyelocytic leukemia / injury / Learning disabilities / disorder / interstitial fibrosis / acid sphingomyelinase deficiency / delayed puberty / severe liver disease / progressive liver dysfunction / illness / end-stage liver disease / MS / Shouval D. Liver failure / lupus / Liver disease / frank cirrhosis / Pick disease / adult Niemann-Pick disease / ataxia / osteopenia / leukemia / epistaxis / valvular heart disease / pneumonia / acute liver failure / progressive pulmonary disease / cancer / clinically significant liver disease / Disease / rare lysosomal storage disease / coronary artery and valvular heart disease / shortness of breath / NiemannPick disease / cholelithiasis NP-B / pancytopenia / pediatric growth restriction / severe systemic disease / chronic oxygen dependence / ataxia / variable degrees / Niemann-Pick disease / fatal neurodegenerative disorder / Subdural hematoma / hyperlipidemia / progressive heart failure / Gaucher disease / pulmonary hypertension / C disease / infiltrative pulmonary disease / pulmonary disease / Attention deficit hyperactivity disorder / progressive neurologic findings / severe thrombocytopenia / Inherited Disease / oxygendependent pulmonary disease / portal hypertension / Gaucher’s disease / heart failure / liver cancer / Autoimmune disease / disease Depression / fulminant liver failure / Psychiatric disease / cardiac disease / osteoporosis / von Willebrand disease / coronary artery disease / chronic hepatitis / deficiency / cough / inflammation / neurologic findings / depression / idiopathic thrombocytopenic purpura / B Niemann Pick disease / interstitial lung disease / fibrosis / Cor pulmonale / / MedicalTreatment bone marrow transplant / blood transfusions / bypass surgery / enzyme replacement therapy / liver transplants / heart surgery / / NaturalFeature Sinai / Vanier MT / Mount Sinai / / Organization Mount Sinai International Center / Mount Sinai’s International Center / National Institute of Health / Mount Sinai School of Medicine / American College of Medical Genetics and Genomics Morbidity / Mount Sinai General Clinical Research Center / National Center for Research Resources / / Person Melissa P. Wasserstein / Petit / Edward H. Schuchman / Margaret M. McGovern / Robert J. Desnick / Natalie Lippa / Emilia Bagiella / / Position Bone Miner / MP / physician / consultant / Major / / ProvinceOrState New York / / Technology Genomics / tomography / transplantation / magnetic resonance imaging / genotype / / SocialTag 
Original Research Article © American College of Medical Genetics and Genomics Morbidity and mortality in type B Niemann–Pick disease Margaret M. McGovern, MD, PhD1, Natalie Lippa, MS, CGC2, Emilia Bagiella, PhD2,