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Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.
Carrillo, Nuria; Malicdan, May C; Leoyklang, Petcharat; Shrader, Joseph A; Joe, Galen; Slota, Christina; Perreault, John; Heiss, John D; Class, Bradley; Liu, Chia-Ying; Bradley, Kennan; Jodarski, Colleen; Ciccone, Carla; Driscoll, Claire; Parks, Rebecca; Van Wart, Scott; Bayman, Levent; Coffey, Christopher S; Quintana, Melanie; Berry, Scott M; Huizing, Marjan; Gahl, William A.
Afiliación
  • Carrillo N; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Malicdan MC; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Leoyklang P; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Shrader JA; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Joe G; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.
  • Slota C; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.
  • Perreault J; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Heiss JD; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Class B; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
  • Liu CY; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Bradley K; Radiology and Imaging Sciences, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.
  • Jodarski C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Ciccone C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Driscoll C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Parks R; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
  • Van Wart S; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.
  • Bayman L; Enhanced Pharmacodynamics LLC, Buffalo, NY, USA.
  • Coffey CS; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA.
  • Quintana M; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA.
  • Berry SM; Berry Consultants LLC, Austin, TX, USA.
  • Huizing M; Berry Consultants LLC, Austin, TX, USA.
  • Gahl WA; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Genet Med ; 23(11): 2067-2075, 2021 11.
Article en En | MEDLINE | ID: mdl-34257421
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Miopatías Distales / Enfermedades Musculares Tipo de estudio: Prognostic_studies Límite: Adult / Humans Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Añadir a Mi BVS
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XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Miopatías Distales / Enfermedades Musculares Tipo de estudio: Prognostic_studies Límite: Adult / Humans Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2021 Tipo del documento: Article País de afiliación: Estados Unidos