Melissa P Wasserstein Selected Research

Niemann-Pick Diseases (Niemann Pick Disease)

9/2021	Plasma lyso-sphingomyelin levels are positively associated with clinical severity in acid sphingomyelinase deficiency.
9/2020	Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment.
1/2018	Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months.
6/2016	Types A and B Niemann-Pick Disease.
1/2016	Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency.
1/2016	Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency).
9/2015	Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency.
3/2015	Types A and B Niemann-Pick disease.
8/2013	Morbidity and mortality in type B Niemann-Pick disease.
8/2012	Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B).

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Melissa P Wasserstein Research Topics

Disease

14	Niemann-Pick Diseases (Niemann Pick Disease) 09/2021 - 07/2004
7	Type B Niemann-Pick Disease 01/2016 - 12/2002
4	Homocystinuria 01/2020 - 12/2009
4	Methylmalonic acidemia 11/2013 - 01/2008
3	Hepatomegaly 09/2020 - 08/2008
3	Fibrosis (Cirrhosis) 09/2020 - 08/2013
2	Splenomegaly 10/2021 - 08/2008
2	Globoid Cell Leukodystrophy (Krabbe Disease) 12/2016 - 03/2016
2	Lung Diseases (Lung Disease) 09/2015 - 08/2013
1	Intellectual Disability (Idiocy) 01/2019
1	Phenylketonurias (Phenylketonuria) 01/2018
1	Infections 06/2016
1	Drug-Related Side Effects and Adverse Reactions 01/2016
1	Acute-Phase Reaction 01/2016
1	Fever (Fevers) 01/2016
1	Inflammation (Inflammations) 01/2016
1	Nausea 01/2016
1	Neurologic Manifestations (Neurological Manifestations) 01/2016
1	Pain (Aches) 01/2016
1	Vomiting 01/2016
1	Type A Niemann-Pick Disease 01/2016
1	Hypersensitivity (Allergy) 09/2015
1	Liver Failure 08/2013
1	Rare Diseases (Rare Disease) 08/2012
1	Propionic Acidemia 02/2010
1	Late Onset Disorders 02/2010
1	Methylmalonic acidemia with homocystinuria 02/2010
1	Inborn Errors Metabolism (Inborn Errors of Metabolism) 07/2009
1	Peripheral Nervous System Diseases (PNS Diseases) 10/2006
1	Liver Diseases (Liver Disease) 12/2004
1	Hypersplenism (Anemia, Splenic) 12/2004
1	Hypertriglyceridemia 07/2004
1	Heart Diseases (Heart Disease) 07/2004
1	Cardiovascular Diseases (Cardiovascular Disease) 07/2004
1	Adrenoleukodystrophy (Adrenoleukodystrophy, X-Linked) 06/2003

Drug/Important Bio-Agent (IBA)

11	neodymium pyrocatechin disulfonate (NPD)IBA 06/2016 - 12/2002
6	LipidsIBA 09/2020 - 07/2004
5	SphingomyelinsIBA 09/2021 - 08/2012
5	olipudase alfaIBA 09/2020 - 09/2015
5	AcidsIBA 01/2018 - 07/2010
5	Sphingomyelin Phosphodiesterase (Sphingomyelinase)IBA 01/2018 - 07/2010
3	Biomarkers (Surrogate Marker)IBA 09/2021 - 08/2012
3	Vitamin B 12 (Cyanocobalamin)FDA LinkGeneric 11/2013 - 12/2009
1	EnzymesIBA 09/2020
1	ChromatinIBA 01/2019
1	Histones (Histone)IBA 01/2019
1	Phenylalanine Hydroxylase (Phenylalanine 4 Monooxygenase)IBA 01/2018
1	pegvaliaseIBA 01/2018
1	Phenylalanine (L-Phenylalanine)FDA Link 01/2018
1	GalactosylceramidaseIBA 03/2016
1	C-Reactive ProteinIBA 01/2016
1	Calcitonin (Calcitonin, Eel)FDA LinkGeneric 01/2016
1	Interleukin-8 (Interleukin 8)IBA 01/2016
1	chitotriosidaseIBA 09/2015
1	AntibodiesIBA 09/2015
1	Oxygen (Dioxygen)IBA 08/2013
1	Methionine (L-Methionine)FDA Link 02/2010
1	propionylcarnitineIBA 02/2010
1	Hydroxocobalamin FDA Link 02/2010
1	Methylmalonic AcidIBA 12/2009
1	HomocysteineIBA 12/2009
1	Aspirin (Acetylsalicylic Acid)FDA LinkGeneric 07/2009
1	Retinaldehyde (Retinal)IBA 10/2006
1	Transaminases (Aminotransferases)IBA 12/2004
1	BilirubinIBA 12/2004
1	Glucose (Dextrose)FDA LinkGeneric 07/2004
1	LDL CholesterolIBA 07/2004
1	HDL CholesterolIBA 07/2004
1	arginine butyrateIBA 06/2003
1	Insulin (Novolin)FDA Link 04/2003

Therapy/Procedure

5	Enzyme Replacement Therapy 01/2018 - 08/2008
2	Therapeutics 08/2013 - 07/2009
1	Splenectomy 10/2021
1	Heart-Assist Devices 01/2020
1	Heart Transplantation (Grafting, Heart) 01/2020
1	Hematopoietic Stem Cell Transplantation 12/2016
1	Transplantation 08/2013
1	Liver Transplantation 01/2008