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Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial.
Koenekoop, Robert K; Sui, Ruifang; Sallum, Juliana; van den Born, L Ingeborgh; Ajlan, Radwan; Khan, Ayesha; den Hollander, Anneke I; Cremers, Frans P M; Mendola, Janine D; Bittner, Ava K; Dagnelie, Gislin; Schuchard, Ronald A; Saperstein, David A.
Affiliation
  • Koenekoop RK; McGill Ocular Genetics Laboratory and Paediatric Ophthalmology, Montreal Children's Hospital, Departments of Paediatric Surgery, Human Genetics and Ophthalmology, McGill University Health Centre, Montreal, Quebec, Canada. Electronic address: robert.koenekoop@mcgill.ca.
  • Sui R; Ophthalmology, Peking Union Medical College Hospital, Beijing, China.
  • Sallum J; Ophthalmology, Federal University of São Paulo, São Paulo, Brazil.
  • van den Born LI; Medical Retina, Rotterdam Eye Hospital, Rotterdam, Netherlands.
  • Ajlan R; McGill Ocular Genetics Laboratory and Paediatric Ophthalmology, Montreal Children's Hospital, Departments of Paediatric Surgery, Human Genetics and Ophthalmology, McGill University Health Centre, Montreal, Quebec, Canada.
  • Khan A; McGill Ocular Genetics Laboratory and Paediatric Ophthalmology, Montreal Children's Hospital, Departments of Paediatric Surgery, Human Genetics and Ophthalmology, McGill University Health Centre, Montreal, Quebec, Canada.
  • den Hollander AI; Department of Ophthalmology, Department of Human Genetics and Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, Netherlands.
  • Cremers FP; Department of Human Genetics and Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, Netherlands.
  • Mendola JD; McGill Vision Research Unit and Department of Ophthalmology, McGill University Health Centre, Montreal, Quebec, Canada.
  • Bittner AK; The Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD, USA.
  • Dagnelie G; The Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD, USA.
  • Schuchard RA; Neurosurgery, Stanford University, CA, USA.
  • Saperstein DA; Vitreoretinal Associates of Washington, Seattle, WA, USA.
Lancet ; 384(9953): 1513-20, 2014 Oct 25.
Article in En | MEDLINE | ID: mdl-25030840
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Vitamin A / Blindness / Leber Congenital Amaurosis Type of study: Observational_studies Limits: Adolescent / Adult / Child / Humans Language: En Journal: Lancet Year: 2014 Document type: Article Country of publication:
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Vitamin A / Blindness / Leber Congenital Amaurosis Type of study: Observational_studies Limits: Adolescent / Adult / Child / Humans Language: En Journal: Lancet Year: 2014 Document type: Article Country of publication: