UshStat (SAR421869) / Oxford Biomedica, Sanofi 
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 1 Disease   1 Trial   1 Trial   38 News 
  • ||||||||||  UshStat (SAR421869) / Oxford Biomedica, Sanofi
    Trial completion date, Trial primary completion date:  Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B (clinicaltrials.gov) -  Jan 18, 2020   
    P1/2,  N=9, Terminated, 
    Trial completion date: Nov 2033 --> Jun 2031 | Trial primary completion date: Nov 2033 --> Jun 2031 Trial completion date: Apr 2017 --> Aug 2019 | Trial primary completion date: Apr 2017 --> Aug 2019
  • ||||||||||  UshStat (SAR421869) / Oxford Biomedica, Sanofi
    Journal:  The Usher Syndrome, a Human Ciliopathy (Pubmed Central) -  Jun 28, 2019   
    Currently, gene-based therapy concepts, such as gene addition, applications of antisense oligonucleotides and TRIDs ("translational readthrough inducing drugs") for the readthrough of nonsense mutations are preclinically evaluated. For USH1B/MYO7A the UshStat gene therapy clinical trial is ongoing.
  • ||||||||||  UshStat (SAR421869) / Oxford Biomedica, Sanofi
    Clinical, Journal:  Genetic screening of Russian Usher syndrome patients toward selection for gene therapy. (Pubmed Central) -  Apr 14, 2019   
    P=N/A
    Clinically the prevalence of USH2 is low (39.28%) and the frequency of MYO7A mutations responsible for USH1B is very high (63.63%, N = 7/11) compared to other cohorts. These seven patients carrying MYO7A mutations are preliminarily eligible for the UshStat® gene therapy.