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Esmeralda Martins Selected Research

vestronidase alfa

5/2024Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.
1/2022Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.
9/2020Corrigendum 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].
1/2020The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII.

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Esmeralda Martins Research Topics

Disease

4Lysosomal Storage Diseases (Lysosomal Storage Disease)
05/2024 - 01/2019
4Mucopolysaccharidosis VII (Sly Syndrome)
05/2024 - 01/2020
4Mitochondrial Diseases (Mitochondrial Disease)
01/2023 - 01/2019
4Phenylketonurias (Phenylketonuria)
01/2021 - 04/2019
3Mucopolysaccharidoses
06/2023 - 01/2020
3Metabolic Diseases (Metabolic Disease)
01/2022 - 06/2011
2Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy)
06/2023 - 01/2019
2Maple Syrup Urine Disease
08/2010 - 06/2008
1Multiple Acyl Coenzyme A Dehydrogenase Deficiency
02/2022
1Hydrops Fetalis (Fetal Edema)
01/2022
1Dyslipidemias (Dyslipidemia)
01/2022
1Cognitive Dysfunction
01/2022
1GM2 Gangliosidoses (GM2 Gangliosidosis)
09/2020
1Rare Diseases (Rare Disease)
01/2020
1Overweight
10/2019
1Non-alcoholic Fatty Liver Disease
10/2019
1Obesity
10/2019
13-Methylglutaconic Aciduria
09/2019
1Gaucher Disease (Gaucher's Disease)
01/2019
1Wolman Disease (Wolman's Disease)
10/2018
1Hemophagocytic Lymphohistiocytosis (Hemophagocytic Syndrome)
10/2018
1Infantile Refsum Disease (Infantile Phytanic Acid Storage Disease)
01/2016
1Protein Deficiency
05/2015
1Peroxisomal Disorders (Peroxisomal Disorder)
05/2015
1Peroxisome biogenesis disorders
05/2015
1Galactosemias (Galactosemia)
01/2014
1Trimethylaminuria
09/2013
1Medium chain acyl CoA dehydrogenase deficiency
07/2013
1Congenital Disorders of Glycosylation
01/2013
1Communication Disorders (Communication Disorder)
07/2011
1Hyperinsulinism (Hyperinsulinemia)
06/2011
1Hypoglycemia (Reactive Hypoglycemia)
06/2011
13-Hydroxyacyl-CoA Dehydrogenase Deficiency
06/2011
1Methylmalonic acidemia
04/2008
1Homocystinuria
04/2008

Drug/Important Bio-Agent (IBA)

5EnzymesIBA
05/2024 - 05/2015
4vestronidase alfaIBA
05/2024 - 01/2020
4Proteins (Proteins, Gene)FDA Link
09/2019 - 01/2013
3GlucuronidaseIBA
05/2024 - 01/2020
3Lysergic Acid Diethylamide (LSD)IBA
11/2023 - 01/2019
3Fatty Acids (Saturated Fatty Acids)IBA
02/2022 - 06/2011
3Phenylalanine (L-Phenylalanine)FDA Link
01/2021 - 04/2019
2Dermatan SulfateIBA
06/2023 - 01/2022
2GlycosaminoglycansIBA
06/2023 - 01/2022
2Carnitine (L-Carnitine)FDA LinkGeneric
02/2022 - 07/2013
2Amino AcidsFDA Link
01/2021 - 01/2021
2LipidsIBA
01/2019 - 01/2013
2Mitochondrial DNA (mtDNA)IBA
01/2019 - 07/2011
23-Hydroxyacyl-CoA DehydrogenaseIBA
05/2015 - 06/2011
1Adenosine Triphosphate (ATP)IBA
01/2023
1acylcarnitineIBA
02/2022
1AcidsIBA
02/2022
1Riboflavin (Vitamin B2)FDA LinkGeneric
02/2022
1Triglycerides (Triacylglycerol)IBA
01/2022
1Lipoproteins (Lipoprotein)IBA
01/2022
1Chondroitin Sulfates (Chondroitin Sulfate)IBA
01/2022
1CholesterolIBA
01/2022
1Heparitin Sulfate (Heparan Sulfate)IBA
01/2022
1Phenylalanine Hydroxylase (Phenylalanine 4 Monooxygenase)IBA
01/2021
1Caseins (Casein)FDA Link
01/2021
1caseinomacropeptideIBA
01/2021
1Hypoglycemic Agents (Hypoglycemics)IBA
01/2021
1FructoseIBA
10/2019
1Alanine Transaminase (SGPT)IBA
10/2019
1IronIBA
09/2019
1SulfurIBA
09/2019
1Mixed Function Oxygenases (Monooxygenases)IBA
06/2019
1Dietary ProteinsIBA
04/2019
1Galactosylceramides (Galactocerebrosides)IBA
01/2019
1AntigensIBA
01/2019
1Sebelipase alfaIBA
10/2018
1Phytanic AcidIBA
01/2016
1galactose-1-phosphateIBA
01/2014
1trimethylamineIBA
09/2013
1OxidesIBA
09/2013
1dimethylaniline monooxygenase (N-oxide forming) (flavin-containing monooxygenase)IBA
09/2013
1Branched-Chain Amino AcidsIBA
08/2010
1Oxidoreductases (Dehydrogenase)IBA
06/2008
1Vitamin B 12 (Cyanocobalamin)FDA LinkGeneric
04/2008

Therapy/Procedure

3Enzyme Replacement Therapy
01/2022 - 01/2020
1Renal Dialysis (Hemodialysis)
02/2022
1Precision Medicine
01/2019