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Paul Harmatz Selected Research

Iron Overload

2/2015Pulmonary hypertension in well-transfused thalassemia major patients.
2/2013Combined chelation therapy with deferasirox and deferoxamine in thalassemia.
4/2012A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload.
2/2012Electrocardiographic consequences of cardiac iron overload in thalassemia major.
4/2011A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload.
10/2010Management of chronic viral hepatitis in patients with thalassemia: recommendations from an international panel.
2/2008Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies.
9/2007Serum ferritin underestimates liver iron concentration in transfusion independent thalassemia patients as compared to regularly transfused thalassemia and sickle cell patients.
6/2007Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expression.
4/2007Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload.
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Paul Harmatz Research Topics

Disease

26Mucopolysaccharidoses
09/2023 - 02/2008
17Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy)
01/2021 - 05/2004
14Iron Overload
02/2015 - 11/2004
13beta-Thalassemia (Cooley's Anemia)
03/2013 - 01/2005
12Thalassemia
02/2013 - 01/2005
11Mucopolysaccharidosis II (Hunter Syndrome)
11/2023 - 08/2006
10Lysosomal Storage Diseases (Lysosomal Storage Disease)
01/2023 - 04/2005
8Sickle Cell Anemia (Hemoglobin S Disease)
08/2009 - 01/2005
7Disease Progression
11/2021 - 04/2005
6Mucopolysaccharidosis I (Hurler Syndrome)
01/2024 - 12/2013
6Achondroplasia
01/2024 - 01/2019
6Mucopolysaccharidosis IV (Morquio Syndrome)
06/2022 - 01/2013
6Inflammation (Inflammations)
03/2013 - 10/2006
5Cognitive Dysfunction
01/2023 - 01/2012
5Hemoglobinopathies
03/2010 - 01/2005
4Mucopolysaccharidosis VII (Sly Syndrome)
09/2020 - 01/2018
4Hemolysis
02/2015 - 01/2005
3Glycogen Storage Disease Type II (Pompe's Disease)
12/2022 - 01/2013
3Spinal Cord Compression
01/2020 - 01/2013
3Hepatitis
10/2010 - 09/2005
3Inflammatory Bowel Diseases (Inflammatory Bowel Disease)
02/2009 - 02/2007
2Medically Unexplained Symptoms
01/2024 - 09/2022
2Mucopolysaccharidosis III (Sanfilippo Syndrome)
01/2022 - 01/2018
2Rare Diseases (Rare Disease)
10/2021 - 01/2020
2Sleep Wake Disorders
01/2019 - 01/2019
2Body Weight (Weight, Body)
01/2019 - 04/2005
2Hypophosphatasia
01/2019 - 01/2017
2alpha-Mannosidosis
11/2018 - 01/2018
2Pain (Aches)
01/2017 - 10/2015
2Gaucher Disease (Gaucher's Disease)
02/2016 - 03/2013
2Anemia
02/2015 - 06/2007
2Siderosis
01/2014 - 10/2010
2Multiple Sulfatase Deficiency Disease (Mucosulfatidosis)
03/2013 - 04/2010
2Cardiomyopathies (Cardiomyopathy)
02/2012 - 01/2009
2Phenylketonurias (Phenylketonuria)
05/2009 - 01/2008
2Neoplasms (Cancer)
02/2009 - 02/2007
2Ulcerative Colitis
02/2009 - 02/2007
2Crohn Disease (Crohn's Disease)
02/2009 - 03/2008
2Obesity
01/2009 - 08/2008
2Cardiovascular Diseases (Cardiovascular Disease)
01/2009 - 08/2008
2Hemosiderosis
09/2005 - 01/2005
1Injection Site Reaction
01/2024
1Bronchiolitis
01/2024
1Otitis Media
01/2024
1Gastroenteritis
01/2024
1Sudden Infant Death (SID)
01/2024
1Vomiting
01/2024
1Erythema
01/2024
1Pneumonia (Pneumonitis)
01/2024
1Paramyxoviridae Infections (Parainfluenza)
01/2024
1Absence Epilepsy (Childhood Absence Epilepsy)
01/2024
1Skull Fractures (Skull Fracture)
01/2024

Drug/Important Bio-Agent (IBA)

28IronIBA
02/2015 - 11/2004
20EnzymesIBA
01/2024 - 06/2005
14N-Acetylgalactosamine-4-Sulfatase (Arylsulfatase B)IBA
01/2019 - 05/2004
12GlycosaminoglycansIBA
01/2024 - 04/2005
10idursulfaseFDA Link
11/2023 - 08/2006
6human GALNS proteinIBA
01/2017 - 11/2014
6Deferoxamine (Desferal)FDA LinkGeneric
02/2013 - 01/2005
5vosoritideIBA
01/2024 - 01/2019
5DeferasiroxFDA Link
02/2013 - 01/2005
4Iduronidase (alpha-L-Iduronidase)IBA
01/2024 - 12/2013
4galsulfaseFDA Link
01/2021 - 08/2009
4vestronidase alfaIBA
09/2020 - 01/2018
4Biomarkers (Surrogate Marker)IBA
01/2018 - 10/2006
4Chelating AgentsIBA
04/2012 - 01/2005
4Ferritins (Ferritin)IBA
02/2008 - 04/2007
3C-Type Natriuretic Peptide (Peptide, C-Type Natriuretic)IBA
01/2024 - 01/2019
3Keratan SulfateIBA
06/2022 - 09/2015
3SulfatasesIBA
02/2016 - 04/2010
2Heparitin Sulfate (Heparan Sulfate)IBA
01/2023 - 01/2022
2RNA (Ribonucleic Acid)IBA
06/2022 - 06/2007
2Type 3 Fibroblast Growth Factor Receptor (Fibroblast Growth Factor Receptor 3)IBA
01/2022 - 12/2021
2GlucuronidaseIBA
01/2020 - 09/2019
2N- (4'- fluorobutyrophenone)- 4- (4- chlorophenyl)pyridinium (HPP(+))IBA
01/2019 - 01/2017
2asfotase alfaIBA
01/2019 - 01/2017
2alpha-Mannosidase (LAMAN)IBA
11/2018 - 01/2018
2AntibodiesIBA
01/2017 - 09/2015
2imigluceraseFDA Link
02/2016 - 03/2013
2Proteins (Proteins, Gene)FDA Link
09/2015 - 01/2008
2Hemoglobins (Hemoglobin)IBA
02/2015 - 03/2010
24,5- dihydro- 2- (2- hydroxy- 3- (3,6,9- trioxadecyloxy)phenyl)- 4- methyl- 4- thiazolecarboxylic acidIBA
04/2012 - 04/2011
2DeferiproneIBA
10/2011 - 01/2005
2Isoflurophate (DFP)FDA Link
10/2011 - 01/2005
2Antiviral Agents (Antivirals)IBA
10/2010 - 08/2008
2Dermatan SulfateIBA
04/2010 - 08/2009
2CreatinineIBA
04/2010 - 04/2005
2HepcidinsIBA
08/2009 - 06/2007
2CytokinesIBA
08/2009 - 03/2008
2Phenylalanine (L-Phenylalanine)FDA Link
05/2009 - 01/2008
2sapropterin (tetrahydrobiopterin)FDA Link
05/2009 - 01/2008
2Folic Acid (Vitamin M)FDA LinkGeneric
02/2009 - 02/2007
2Ribavirin (Virazole)FDA LinkGeneric
08/2008 - 01/2005
2Interferon-alpha (Interferon Alfa)IBA
08/2008 - 01/2005
1Transferrin Receptors (Transferrin Receptor)IBA
01/2024

Therapy/Procedure

35Enzyme Replacement Therapy
01/2024 - 05/2004
22Therapeutics
01/2024 - 01/2005
4Chelation Therapy (Therapy, Chelation)
02/2013 - 01/2005
4Blood Transfusion (Blood Transfusions)
04/2010 - 01/2005
2Hematopoietic Stem Cell Transplantation
01/2020 - 01/2019