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A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome.
Abreu, Damien; Stone, Stephen I; Pearson, Toni S; Bucelli, Robert C; Simpson, Ashley N; Hurst, Stacy; Brown, Cris M; Kries, Kelly; Onwumere, Chinyere; Gu, Hongjie; Hoekel, James; Tychsen, Lawrence; Van Stavern, Gregory P; White, Neil H; Marshall, Bess A; Hershey, Tamara; Urano, Fumihiko.
Afiliación
  • Abreu D; Division of Endocrinology, Metabolism, and Lipid Research, Department of Medicine.
  • Stone SI; Medical Scientist Training Program.
  • Pearson TS; Division of Endocrinology and Diabetes, Department of Pediatrics.
  • Bucelli RC; Department of Neurology.
  • Simpson AN; Department of Neurology.
  • Hurst S; Center for Clinical Studies.
  • Brown CM; Division of Endocrinology, Metabolism, and Lipid Research, Department of Medicine.
  • Kries K; Division of Endocrinology, Metabolism, and Lipid Research, Department of Medicine.
  • Onwumere C; Division of Endocrinology, Metabolism, and Lipid Research, Department of Medicine.
  • Gu H; Division of Endocrinology, Metabolism, and Lipid Research, Department of Medicine.
  • Hoekel J; Division of Biostatistics.
  • Tychsen L; Department of Ophthalmology & Visual Sciences.
  • Van Stavern GP; Department of Ophthalmology & Visual Sciences.
  • White NH; Department of Ophthalmology & Visual Sciences.
  • Marshall BA; Division of Endocrinology and Diabetes, Department of Pediatrics.
  • Hershey T; Division of Endocrinology and Diabetes, Department of Pediatrics.
  • Urano F; Departments of Psychiatry and Radiology, and.
JCI Insight ; 6(15)2021 08 09.
Article en En | MEDLINE | ID: mdl-34185708
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Calidad de Vida / Síndrome de Wolfram / Agudeza Visual / Interleucina-18 / Dantroleno / Células Secretoras de Insulina / Interleucina-1beta Tipo de estudio: Diagnostic_studies / Observational_studies / Prognostic_studies Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Humans Idioma: En Revista: JCI Insight Año: 2021 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Calidad de Vida / Síndrome de Wolfram / Agudeza Visual / Interleucina-18 / Dantroleno / Células Secretoras de Insulina / Interleucina-1beta Tipo de estudio: Diagnostic_studies / Observational_studies / Prognostic_studies Aspecto: Patient_preference Límite: Adolescent / Adult / Child / Humans Idioma: En Revista: JCI Insight Año: 2021 Tipo del documento: Article