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Continued improvement in disease manifestations of acid sphingomyelinase deficiency for adults with up to 2 years of olipudase alfa treatment: open-label extension of the ASCEND trial.
Wasserstein, Melissa P; Lachmann, Robin; Hollak, Carla; Barbato, Antonio; Gallagher, Renata C; Giugliani, Roberto; Guelbert, Norberto Bernardo; Hennermann, Julia B; Ikezoe, Takayuki; Lidove, Olivier; Mabe, Paulina; Mengel, Eugen; Scarpa, Maurizio; Senates, Ebubekir; Tchan, Michel; Villarrubia, Jesus; Thurberg, Beth L; Yarramaneni, Abhimanyu; Armstrong, Nicole M; Kim, Yong; Kumar, Monica.
Afiliación
  • Wasserstein MP; Children's Hospital at Montefiore and the Albert Einstein College of Medicine, 3411 Wayne Ave, 9th Floor, Bronx, NY, 10467, USA. melissa.wasserstein@einsteinmed.edu.
  • Lachmann R; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
  • Hollak C; Department of Endocrinology and Metabolism, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
  • Barbato A; Department of Clinical Medicine and Surgery, University of Naples "Federico II", Naples, Italy.
  • Gallagher RC; Department of Pediatrics, The University of California San Francisco, San Francisco, CA, USA.
  • Giugliani R; Postgraduate Program in Genetics and Molecular Biology, Med Genet Serv & DR Brasil, HCPA, INAGEMP, DASA, and Casa Dos Raros, UFRGS, Porto Alegre, Brazil.
  • Guelbert NB; Reina Fabiola University Clinic, Córdoba, Argentina.
  • Hennermann JB; Villa Metabolica, Center for Pediatric and Adolescent Medicine, University Medical Center, Mainz, Germany.
  • Ikezoe T; Department of Hematology, Fukushima Medical University, Fukushima, Japan.
  • Lidove O; Department of Internal Medicine, La Croix St Simon Hospital, Paris, France.
  • Mabe P; Clinica Santa Maria, Santiago, Chile.
  • Mengel E; Clinical Science for LSD, SpinCS, Hochheim, Germany.
  • Scarpa M; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, 33100, Udine, Italy.
  • Senates E; Istanbul Medeniyet University, Istanbul, Turkey.
  • Tchan M; Department of Genetic Medicine, Westmead Hospital, Sydney, Australia.
  • Villarrubia J; Hematology Department, Hospital Universitario Ramón y Cajal, Madrid, Spain.
  • Thurberg BL; Sanofi, Cambridge, MA, USA.
  • Yarramaneni A; Sanofi, Bridgewater, NJ, USA.
  • Armstrong NM; Sanofi, Cambridge, MA, USA.
  • Kim Y; Sanofi, Paris, France.
  • Kumar M; Sanofi, Bridgewater, NJ, USA.
Orphanet J Rare Dis ; 18(1): 378, 2023 Dec 02.
Article en En | MEDLINE | ID: mdl-38042851
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades de Niemann-Pick / Enfermedad de Niemann-Pick Tipo A Límite: Adult / Humans Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades de Niemann-Pick / Enfermedad de Niemann-Pick Tipo A Límite: Adult / Humans Idioma: En Revista: Orphanet J Rare Dis Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido