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Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS).
Tjeertes, Jorrit; Bacino, Carlos A; Bichell, Terry Jo; Bird, Lynne M; Bustamante, Mariana; Crean, Rebecca; Jeste, Shafali; Komorowski, Robert W; Krishnan, Michelle L; Miller, Meghan T; Nobbs, David; Ochoa-Lubinoff, Cesar; Parkerson, Kimberly A; Rotenberg, Alexander; Sadhwani, Anjali; Shen, Mark D; Squassante, Lisa; Tan, Wen-Hann; Vincenzi, Brenda; Wheeler, Anne C; Hipp, Joerg F; Berry-Kravis, Elizabeth.
Afiliação
  • Tjeertes J; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland. jorrit.tjeertes@roche.com.
  • Bacino CA; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
  • Bichell TJ; Texas Children's Hospital, Houston, TX, USA.
  • Bird LM; COMBINEDBrain, Brentwood, TN, USA.
  • Bustamante M; Department of Pediatrics, University of California San Diego, San Diego, CA, USA.
  • Crean R; Division of Dysmorphology/Genetics, Rady Children's Hospital, San Diego, CA, USA.
  • Jeste S; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Komorowski RW; Ionis Pharmaceuticals Inc, Carlsbad, CA, USA.
  • Krishnan ML; Children's Hospital Los Angeles, Los Angeles, CA, USA.
  • Miller MT; Keck School of Medicine of USC, Los Angeles, CA, USA.
  • Nobbs D; Ionis Pharmaceuticals Inc, Carlsbad, CA, USA.
  • Ochoa-Lubinoff C; , Biogen, Cambridge, MA, USA.
  • Parkerson KA; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Rotenberg A; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Sadhwani A; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Shen MD; Departments of Pediatrics, Division of Developmental-Behavioral Pediatrics, Rush University Medical Center, Chicago, IL, USA.
  • Squassante L; , Biogen, Cambridge, MA, USA.
  • Tan WH; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Vincenzi B; Department of Psychiatry and Behavioral Services, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Wheeler AC; Carolina Institute for Developmental Disabilities & UNC Neuroscience Center, University of North Carolina, Chapel Hill, NC, USA.
  • Hipp JF; F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070, Basel, Switzerland.
  • Berry-Kravis E; Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
J Neurodev Disord ; 15(1): 22, 2023 07 26.
Article em En | MEDLINE | ID: mdl-37495977

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Angelman / COVID-19 Tipo de estudo: Guideline / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Revista: J Neurodev Disord Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Suíça

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Angelman / COVID-19 Tipo de estudo: Guideline / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Humans Idioma: En Revista: J Neurodev Disord Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Suíça