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  • KOLs
  • Lysosomal Storage

Lysosomal Storage



J John Hopwood #1
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
Robert J Desnick #2
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

Roscoe O Brady #3
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
Elizabeth F Neufeld #4
Elizabeth F Neufeld
Department of Biological Chemistry, David Geffen School of M
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Known for Lysosomal Enzyme | Mouse Model | Iduronate Sulfatase | Cultured Fibroblasts
Frances M Platt #5
Frances M Platt
Department of Pharmacology, University of Oxford, Oxford, UK
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Known for Sandhoff Disease | Lysosomal Storage | Fusion Pore | Substrate Reduction Therapy
MARIE THERESE VANIER #6
MARIE THERESE VANIER
Biochimie métabolique, hospices civils de Lyon - HCL, Lyon |
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Known for Pick Disease | Globoid Cell | Mouse Model | Fatty Acid
Steven U Walkley #7
Steven U Walkley
Department of Neuroscience, Rose F. Kennedy Intellectual and
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Known for Ectopic Dendrites | Batten Disease | Cortical Pyramidal Neurons | Lysosomal Storage
Konrad Sandhoff #8
Konrad Sandhoff
Membrane Biology and Lipid Biochemistry Unit, LIMES Institut
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Known for Acid Sphingomyelinase | Ganglioside Gm2 | Gaucher Disease | Rat Liver
Michael H Beck #9
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
Raphael Schiffmann #10
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