Nathalie Guffon Selected Research

olipudase alfa

12/2022	Long-term safety and clinical outcomes of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency: two-year results.
10/2022	One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.
1/2021	One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency.

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Nathalie Guffon Research Topics

Disease

10	Mucopolysaccharidosis II (Hunter Syndrome) 11/2023 - 03/2008
9	Mucopolysaccharidosis I (Hurler Syndrome) 10/2023 - 07/2007
9	Fabry Disease (Fabry's Disease) 01/2023 - 06/2002
9	Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy) 01/2022 - 04/2005
7	Lysosomal Storage Diseases (Lysosomal Storage Disease) 12/2022 - 11/2003
7	Mucopolysaccharidoses 12/2022 - 03/2007
5	alpha-Mannosidosis 01/2023 - 01/2018
4	Niemann-Pick Diseases (Niemann Pick Disease) 12/2022 - 01/2021
4	Hyperammonemia 09/2016 - 04/2005
3	Neurologic Manifestations (Neurological Manifestations) 06/2020 - 01/2018
3	Disease Progression 01/2017 - 04/2005
3	Inborn Urea Cycle Disorders 09/2016 - 12/2012
2	Cognitive Dysfunction 09/2022 - 09/2022
2	Pain (Aches) 01/2022 - 01/2006
2	Type C Niemann-Pick Disease (Niemann Pick Disease, Type C) 01/2017 - 02/2009
2	Ketosis 01/2017 - 05/2011
2	Propionic Acidemia 03/2016 - 01/2008
2	Methylmalonic acidemia 03/2016 - 01/2008
1	Splenomegaly 12/2022
1	Medically Unexplained Symptoms 09/2022
1	Mucopolysaccharidosis IV (Morquio Syndrome) 09/2022
1	Exanthema (Rash) 01/2021
1	Urticaria (Hives) 01/2021
1	Anaphylaxis (Anaphylactic Shock) 01/2021
1	Hypersensitivity (Allergy) 01/2020
1	Inguinal Hernia (Direct Inguinal Hernia) 09/2019
1	Respiratory Tract Infections (Respiratory Tract Infection) 09/2019
1	Mucopolysaccharidosis VII (Sly Syndrome) 09/2019
1	Coxa Valga 09/2019
1	Dysostoses 01/2019
1	Homocystinuria 01/2019
1	Tyrosinemias (Tyrosinemia) 01/2018
1	GM2 Gangliosidoses (GM2 Gangliosidosis) 01/2017
1	Hydrops Fetalis (Fetal Edema) 01/2017
1	Gaucher Disease (Gaucher's Disease) 01/2017
1	Sphingolipidoses (Sphingolipidosis) 01/2017
1	Gastroenteritis 09/2016
1	Metabolic Diseases (Metabolic Disease) 09/2016
1	Hyperargininemia 09/2016
1	Hypercholesterolemia 02/2016

Drug/Important Bio-Agent (IBA)

11	EnzymesIBA 12/2022 - 05/2007
7	GlycosaminoglycansIBA 10/2023 - 04/2005
6	idursulfase FDA Link 11/2023 - 10/2010
6	N-Acetylgalactosamine-4-Sulfatase (Arylsulfatase B)IBA 08/2014 - 04/2006
5	agalsidase beta FDA Link 01/2023 - 01/2006
5	alpha-Galactosidase (Beano)IBA 04/2008 - 06/2002
4	Iduronidase (alpha-L-Iduronidase)IBA 10/2022 - 07/2007
4	alpha-Mannosidase (LAMAN)IBA 06/2020 - 01/2018
3	olipudase alfaIBA 12/2022 - 01/2021
3	AcidsIBA 12/2022 - 03/2016
3	globotriaosylceramide (globotriosylceramide)IBA 05/2015 - 05/2007
2	GalactosidasesIBA 01/2023 - 05/2015
2	Sphingomyelin Phosphodiesterase (Sphingomyelinase)IBA 12/2022 - 02/2022
2	human GALNS proteinIBA 09/2022 - 02/2015
2	Carbamyl Phosphate (Carbamoyl Phosphate)IBA 09/2016 - 01/2008
2	Ligases (Synthetase)IBA 09/2016 - 01/2008
2	4-phenylbutyric acid (Buphenyl)FDA Link 04/2016 - 12/2012
2	galsulfase FDA Link 08/2014 - 03/2014
2	miglustat (Zavesca)FDA Link 11/2011 - 02/2009
2	Glutamic Acid (Glutamate)FDA Link 01/2008 - 08/2005
2	N-acetyl glutamate synthetase deficiencyIBA 08/2005 - 04/2005
1	OligosaccharidesIBA 01/2023
1	Mannose (D-Mannose)IBA 01/2023
1	amsonic acid (DAS)IBA 09/2022
1	LipidsIBA 02/2022
1	SphingomyelinsIBA 02/2022
1	odiparcilIBA 01/2022
1	Alanine Transaminase (SGPT)IBA 01/2021
1	Immunoglobulin G (IgG)IBA 01/2020
1	GlucuronidaseIBA 09/2019
1	Betaine (C.B.B.)FDA Link 01/2019
1	HomocysteineIBA 01/2019
1	nitisinone (Orfadin)FDA Link 01/2018
1	sphingosine phosphorylcholineIBA 01/2017
1	Biomarkers (Surrogate Marker)IBA 01/2017
1	SphingolipidsIBA 01/2017
1	GangliosidesIBA 01/2017
1	Glucosylceramidase (Glucocerebrosidase)IBA 01/2017
1	lysoGM1 gangliosideIBA 01/2017
1	Beta ketothiolase deficiencyIBA 01/2017
1	Ornithine Carbamoyltransferase (Ornithine Transcarbamylase)IBA 09/2016
1	LyasesIBA 09/2016
1	SodiumIBA 09/2016
1	Benzoates (Benzoate)IBA 09/2016
1	Urea (Carbamide)FDA LinkGeneric 09/2016
1	HHH syndromeIBA 09/2016
1	Succinic Acid (Succinate)IBA 09/2016
1	Sodium Benzoate FDA Link 09/2016
1	N-acetylglutamic acidIBA 03/2016
1	Alkaline PhosphataseIBA 02/2016
1	Transaminases (Aminotransferases)IBA 02/2016
1	CopperIBA 02/2016

Therapy/Procedure

21	Enzyme Replacement Therapy 12/2022 - 07/2004
7	Therapeutics 01/2019 - 08/2002
1	Enzyme Therapy 06/2020
1	Osteotomy 09/2019
1	Orthopedic Procedures 09/2019
1	Activities of Daily Living (ADL) 01/2019
1	Cell Transplantation 11/2018
1	Liver Transplantation 02/2016