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Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial.
Schiffmann, Raphael; Cox, Timothy M; Dedieu, Jean-François; Gaemers, Sebastiaan J M; Hennermann, Julia B; Ida, Hiroyuki; Mengel, Eugen; Minini, Pascal; Mistry, Pramod; Musholt, Petra B; Scott, David; Sharma, Jyoti; Peterschmitt, M Judith.
Afiliación
  • Schiffmann R; Institute of Metabolic Disease, Baylor Scott and White Research Institute, Dallas, TX 75246, USA.
  • Cox TM; Department of Medicine, University of Cambridge and Addenbrooke's Hospital, Cambridge CB2 0QQ, UK.
  • Dedieu JF; Neurology Development, Sanofi, 91385 Chilly-Mazarin, France.
  • Gaemers SJM; Global Pharmacovigilance, Sanofi, Amsterdam, 1105 BP, The Netherlands.
  • Hennermann JB; Center for Pediatric and Adolescent Medicine Villa Metabolica, University Medical Center Mainz, 55131 Mainz, Germany.
  • Ida H; Department of Pediatrics, The Jikei University School of Medicine, Tokyo 105-8461, Japan.
  • Mengel E; Center for Pediatric and Adolescent Medicine Villa Metabolica, University Medical Center Mainz, 55131 Mainz, Germany.
  • Minini P; Clinical Science for LSD, SphinCS, 65239 Hochheim, Germany.
  • Mistry P; Biostatistics and Programming, Sanofi, 91385 Chilly-Mazarin, France.
  • Musholt PB; Yale Lysosomal Disease Center and Gaucher Disease Treatment Center, Yale School of Medicine, New Haven, CT 06510, USA.
  • Scott D; R&D Global Development, Sanofi, 65926 Frankfurt, Germany.
  • Sharma J; Medical and Scientific Affairs, Neuroscience, Clario, San Mateo, CA 94404, USA.
  • Peterschmitt MJ; Pharmacokinetics, Dynamics and Metabolism, Sanofi, Bridgewater, NJ 08807, USA.
Brain ; 146(2): 461-474, 2023 02 13.
Article en En | MEDLINE | ID: mdl-36256599
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Gaucher / Enfermedades del Sistema Nervioso Límite: Adult / Humans Idioma: En Revista: Brain Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Enfermedad de Gaucher / Enfermedades del Sistema Nervioso Límite: Adult / Humans Idioma: En Revista: Brain Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos