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  • KOLs
  • Enzyme Replacement Therapy

Enzyme Replacement Therapy



Michael H Beck #1
Michael H Beck
SphinCS, Hochheim, Germany | SphinCS GmbH, Institute Clinica
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Known for Enzyme Replacement Therapy | Agalsidase Alfa | Hunter Syndrome | Fabry Disease
Roscoe O Brady #2
Roscoe O Brady
Department of Neurology, Berenson-Allen Center for Noninvasi
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Known for Enzyme Replacement Therapy | Bipolar Disorder | Bone Marrow | Gene Transfer

Gregory M Pastores #3
Gregory M Pastores
National Centre for Inherited Metabolic Disorders, The Mater
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Known for Enzyme Replacement Therapy | Agalsidase Alfa | Type 1 | Lysosomal Storage Disorders
Robert J Desnick #4
Robert J Desnick
Mount Sinai School of Medicine, New York, New York, United S
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Known for Acute Intermittent Porphyria | Prenatal Diagnosis | Enzyme Replacement Therapy | Ashkenazi Jewish
Nathalie Guffon #5
Nathalie Guffon
Centre de Référence des Maladies Métaboliques, Femme Mère En
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Known for Velmanase Alfa | Fabry Disease | Carglumic Acid | Mucopolysaccharidosis Type
Raphael Schiffmann #6
Raphael Schiffmann
4D Molecular Therapeutics, Inc., Emeryville, CA, USA | Natio
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Known for Mucolipidosis Type | Enzyme Replacement Therapy | Xeroderma Pigmentosum | Fabry Disease
J John Hopwood #7
J John Hopwood
The University of Adelaide, School of Medicine, North Terrac
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Known for Lysosomal Storage | Mps Iiia | Enzyme Replacement Therapy | Sanfilippo Syndrome
Christine M Eng #8
Christine M Eng
Baylor College of Medicine, Houston, TX, USA | Baylor Geneti
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Known for Exome Sequencing | Hunter Syndrome | Fabry Disease | Prenatal Diagnosis
Roberto Giugliani #9
Roberto Giugliani
DASA, São Paulo, Brazil | Universidade Federal do Rio Grande
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Known for Mucopolysaccharidosis Type | Enzyme Replacement Therapy | Hunter Syndrome | Latin America
Ans Tjitske Van der Ploeg #10
Ans Tjitske Van der Ploeg
Center for Lysosomal and Metabolic Diseases, Erasmus MC, Uni
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Known for Glycogen Storage | Muscle Strength | Pompe Disease | Alglucosidase Alfa