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Results 1 - 9 of 9 for Trials
  1. Leber congenital amaurosis 
    ... congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions. J AAPOS. 2009 Dec; ... s congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009 Nov 7;374(9701):1597-605. ...
    https://medlineplus.gov/.../condition/leber-congenital-amaurosis - Genetics
  2. Nearsightedness 
    ... Hosseini SM, Paterson AD; Diabetes Control and Complications Trial/Epidemiology of Diabetes Interventions, and Complications Research Group; ... Ong RT, Doring A; Diabetes Control and Complications Trial/Epidemiology of Diabetes Interventions and Complications (DCCT/EDIC) ...
    https://medlineplus.gov/genetics/condition/nearsightedness - Genetics
  3. Lennox-Gastaut syndrome 
    ... a consensus approach on diagnosis, assessment, management, and trial methodology. Lancet Neurol. 2009 Jan;8(1):82- ...
    https://medlineplus.gov/genetics/condition/lennox-gastaut-syndrome - Genetics
  4. GRN-related frontotemporal lobar degeneration 
    ... frontotemporal dementia: NIH-EXAMINER as a potential clinical trial endpoint. Alzheimers Dement. 2020 Jan;16(1):11- ...
    https://medlineplus.gov/...related-frontotemporal-lobar-degeneration - Genetics
  5. Niemann-Pick disease 
    ... A Niemann-Pick disease: possible endpoints for therapeutic trials. Neurology. 2006 Jan 24;66(2):228-32. ...
    https://medlineplus.gov/genetics/condition/niemann-pick-disease - Genetics
  6. Dentatorubral-pallidoluysian atrophy 
    ... natural history and developing biomarkers to accelerate therapeutic trials in a globally rare repeat expansion disorder. J ...
    https://medlineplus.gov/.../dentatorubral-pallidoluysian-atrophy - Genetics
  7. Proopiomelanocortin deficiency 
    ... to proopiomelanocortin deficiency: three new cases and treatment trials with thyroid hormone and ACTH4-10. J Clin ...
    https://medlineplus.gov/.../condition/proopiomelanocortin-deficiency - Genetics
  8. Age-related macular degeneration 
    ... WL; Complications of Age-Related Macular Degeneration Prevention Trial Research Group; Brucker AJ, Orlin A, Brown G, ...
    https://medlineplus.gov/.../age-related-macular-degeneration - Genetics
  9. Cryopyrin-associated periodic syndromes 
    ... HJ, Touitou I, Ruperto N; Paediatric Rheumatology International Trials Organisation (PRINTO) and the AIDs Delphi study participants. ...
    https://medlineplus.gov/.../cryopyrin-associated-periodic-syndromes - Genetics