Paul Harmatz Selected Research

Enzyme Replacement Therapy

1/2021	Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy.
9/2020	Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].
1/2020	Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.
1/2020	The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.
1/2019	Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance.
1/2019	Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance.
1/2018	Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis.
1/2017	Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study.
2/2016	Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.
9/2015	Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.

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Paul Harmatz Research Topics

Disease

22	Mucopolysaccharidoses 06/2022 - 02/2008
17	Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy) 01/2021 - 05/2004
14	Iron Overload 02/2015 - 11/2004
13	beta-Thalassemia (Cooley's Anemia) 03/2013 - 01/2005
12	Thalassemia 02/2013 - 01/2005
8	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2022 - 04/2005
8	Sickle Cell Anemia (Hemoglobin S Disease) 08/2009 - 01/2005
7	Disease Progression 11/2021 - 04/2005
7	Mucopolysaccharidosis II (Hunter Syndrome) 10/2021 - 08/2006
6	Mucopolysaccharidosis IV (Morquio Syndrome) 06/2022 - 01/2013
6	Inflammation (Inflammations) 03/2013 - 10/2006
5	Hemoglobinopathies 03/2010 - 01/2005
4	Achondroplasia 01/2022 - 01/2019
4	Mucopolysaccharidosis VII (Sly Syndrome) 09/2020 - 01/2018
4	Mucopolysaccharidosis I (Hurler Syndrome) 01/2020 - 12/2013
4	Hemolysis 02/2015 - 01/2005
3	Rare Diseases (Rare Disease) 10/2021 - 01/2018
3	Spinal Cord Compression 01/2020 - 01/2013
3	Hepatitis 10/2010 - 09/2005
3	Inflammatory Bowel Diseases (Inflammatory Bowel Disease) 02/2009 - 02/2007
2	Mucopolysaccharidosis III (Sanfilippo Syndrome) 01/2022 - 01/2018
2	Cognitive Dysfunction 01/2020 - 01/2012
2	Sleep Wake Disorders 01/2019 - 01/2019
2	Hypophosphatasia 01/2019 - 01/2017
2	Body Weight (Weight, Body) 01/2019 - 04/2005
2	alpha-Mannosidosis 11/2018 - 01/2018
2	Hypersensitivity (Allergy) 01/2017 - 05/2004
2	Pain (Aches) 01/2017 - 10/2015
2	Gaucher Disease (Gaucher's Disease) 02/2016 - 03/2013
2	Glycogen Storage Disease Type II (Pompe's Disease) 09/2015 - 01/2013
2	Anemia 02/2015 - 06/2007
2	Siderosis 01/2014 - 10/2010
2	Multiple Sulfatase Deficiency Disease (Mucosulfatidosis) 03/2013 - 04/2010
2	Cardiomyopathies (Cardiomyopathy) 02/2012 - 01/2009
2	Phenylketonurias (Phenylketonuria) 05/2009 - 01/2008
2	Neoplasms (Cancer) 02/2009 - 02/2007
2	Crohn Disease (Crohn's Disease) 02/2009 - 03/2008
2	Ulcerative Colitis 02/2009 - 02/2007
2	Cardiovascular Diseases (Cardiovascular Disease) 01/2009 - 08/2008
2	Obesity 01/2009 - 08/2008
2	Hemosiderosis 09/2005 - 01/2005
1	Type C Niemann-Pick Disease (Niemann Pick Disease, Type C) 11/2021
1	Progressive Myoclonic Epilepsies (Progressive Myoclonic Epilepsy) 01/2020
1	Hydrocephalus (Hydrocephaly) 01/2020

Drug/Important Bio-Agent (IBA)

28	IronIBA 02/2015 - 11/2004
17	EnzymesIBA 01/2020 - 06/2005
14	N-Acetylgalactosamine-4-Sulfatase (Arylsulfatase B)IBA 01/2019 - 05/2004
10	GlycosaminoglycansIBA 06/2022 - 04/2005
6	idursulfase FDA Link 10/2021 - 08/2006
6	human GALNS proteinIBA 01/2017 - 11/2014
6	Deferoxamine (Desferal)FDA LinkGeneric 02/2013 - 01/2005
5	Pharmaceutical PreparationsIBA 01/2018 - 05/2004
5	Deferasirox FDA Link 02/2013 - 01/2005
4	galsulfase FDA Link 01/2021 - 08/2009
4	vestronidase alfaIBA 09/2020 - 01/2018
4	Biomarkers (Surrogate Marker)IBA 01/2018 - 10/2006
4	Chelating AgentsIBA 04/2012 - 01/2005
4	Ferritins (Ferritin)IBA 02/2008 - 04/2007
3	Keratan SulfateIBA 06/2022 - 09/2015
3	vosoritideIBA 12/2021 - 01/2019
3	Iduronidase (alpha-L-Iduronidase)IBA 01/2020 - 12/2013
3	SulfatasesIBA 02/2016 - 04/2010
2	RNA (Ribonucleic Acid)IBA 06/2022 - 06/2007
2	Type 3 Fibroblast Growth Factor Receptor (Fibroblast Growth Factor Receptor 3)IBA 01/2022 - 12/2021
2	C-Type Natriuretic Peptide (Peptide, C-Type Natriuretic)IBA 12/2021 - 01/2019
2	GlucuronidaseIBA 01/2020 - 09/2019
2	N- (4'- fluorobutyrophenone)- 4- (4- chlorophenyl)pyridinium (HPP(+))IBA 01/2019 - 01/2017
2	asfotase alfaIBA 01/2019 - 01/2017
2	alpha-Mannosidase (LAMAN)IBA 11/2018 - 01/2018
2	AntibodiesIBA 01/2017 - 09/2015
2	imiglucerase FDA Link 02/2016 - 03/2013
2	Proteins (Proteins, Gene)FDA Link 09/2015 - 01/2008
2	Hemoglobins (Hemoglobin)IBA 02/2015 - 03/2010
2	4,5- dihydro- 2- (2- hydroxy- 3- (3,6,9- trioxadecyloxy)phenyl)- 4- methyl- 4- thiazolecarboxylic acidIBA 04/2012 - 04/2011
2	DeferiproneIBA 10/2011 - 01/2005
2	Isoflurophate (DFP)FDA Link 10/2011 - 01/2005
2	Antiviral Agents (Antivirals)IBA 10/2010 - 08/2008
2	CreatinineIBA 04/2010 - 04/2005
2	Dermatan SulfateIBA 04/2010 - 08/2009
2	HepcidinsIBA 08/2009 - 06/2007
2	CytokinesIBA 08/2009 - 03/2008
2	Phenylalanine (L-Phenylalanine)FDA Link 05/2009 - 01/2008
2	sapropterin (tetrahydrobiopterin)FDA Link 05/2009 - 01/2008
2	Folic Acid (Vitamin M)FDA LinkGeneric 02/2009 - 02/2007
2	Interferon-alpha (Interferon Alfa)IBA 08/2008 - 01/2005
2	Ribavirin (Virazole)FDA LinkGeneric 08/2008 - 01/2005
1	Protein Isoforms (Isoforms)IBA 06/2022
1	Heparitin Sulfate (Heparan Sulfate)IBA 01/2022
1	N-sulfoglucosamine sulfohydrolase (sulfamidase)IBA 01/2022
1	Tyrosine Kinase InhibitorsIBA 01/2022
1	infigratinibIBA 01/2022
1	arimoclomolIBA 11/2021
1	CeramidasesIBA 01/2020

Therapy/Procedure

30	Enzyme Replacement Therapy 01/2021 - 05/2004
17	Therapeutics 01/2022 - 01/2005
4	Chelation Therapy (Therapy, Chelation) 02/2013 - 01/2005
4	Blood Transfusion (Blood Transfusions) 04/2010 - 01/2005
2	Hematopoietic Stem Cell Transplantation 01/2020 - 01/2019
1	Fetal Therapies (Fetal Therapy) 01/2022
1	Intravenous Injections 01/2022