Mucopolysaccharidosis: Top KOLs in the world #1 J John Hopwood The University of Adelaide, School of Medicine, North Terrac star star star star star Known for Lysosomal Storage | Mps Iiia | Enzyme Replacement Therapy | Sanfilippo Syndrome | Mucopolysaccharidosis Type
#2 Roberto Giugliani DASA, São Paulo, Brazil | Universidade Federal do Rio Grande star star star star star Known for Mucopolysaccharidosis Type | Enzyme Replacement Therapy | Hunter Syndrome | Latin America | Fabry Disease
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#3 Michael H Beck SphinCS, Hochheim, Germany | SphinCS GmbH, Institute Clinica star star star star star_half Known for Enzyme Replacement Therapy | Agalsidase Alfa | Hunter Syndrome | Fabry Disease | Allergic Contact Dermatitis
#4 James Edmond Wraith Willink Biochemical Genetics Department, St. Mary’s Hospital star star star star star_half Known for Hurler Syndrome | Patients Mps | Hunter Outcome Survey | Enzyme Replacement Therapy | Mucopolysaccharidosis Type
#5 Joseph Muenzer University of North Carolina Children's Hospital, Chapel Hil star star star star star_border Known for Enzyme Replacement Therapy | Mucopolysaccharidosis Type | North Carolina | Patients Mps | Hunter Syndrome
#6 Paul R Harmatz University of California San Francisco, San Francisco, CA, U star star star star star_border Known for Hunter Syndrome | Iron Overload | Elosulfase Alfa | Mucopolysaccharidosis Type | Sickle Cell Disease
#7 Elizabeth F Neufeld Department of Biological Chemistry, David Geffen School of M star star star star star_border Known for Lysosomal Enzyme | Mouse Model | Iduronate Sulfatase | Cultured Fibroblasts | Hunter Syndrome
#8 Chester B Whitley Gene Therapy Center Department of Pediatrics University of M star star star star star_border Known for Enzyme Replacement Therapy | Fabry Disease | Gene Transfer | Mucopolysaccharidosis Type | Hurler Syndrome
#9 Nathalie Guffon Centre de Référence des Maladies Métaboliques, Femme Mère En star star star star star_border Known for Velmanase Alfa | Fabry Disease | Carglumic Acid | Mucopolysaccharidosis Type | Enzyme Replacement Therapy
#10 Emil D Kakkis Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 9 star star star star star_border Known for Mps Vii | Vestronidase Alfa | Gne Myopathy | Immune Tolerance | Enzyme Replacement Therapy
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Recently Asked Questions
Mucopolysaccharidosis Mucopolysaccharidosis (MPS) is a group of rare, inherited metabolic disorders that affect many different tissues and organs in the body. People with MPS...