Queen Square Centre for Neuromuscular Diseases


Translational research conference 2016

1 March 2016


indigo-sandbox.ucl.ac.uk/cnmd/events/translational-research-conference" target="_self">translational research conference returns this year in March, on the 22nd and 23rd.

Sessions will include presentations and discussion on:

Day 1

  • Genomic Therapies
  • Activating internal ribosome entry site in Duchenne muscular dystrophy / dystrophin 5
  • From pathogenesis to therapy in spinal muscular atrophy
  • Targeting 3'end elements involved in mRNA processing is an efficient therapeutic strategy for FacioScapuloHumeral Dystrophy: a new gene silencing approach
  • Next generation biomarkers
  • Next generation in vivo imaging technologies in DMD -the BIOIMAGE-NMD programme
  • MRI Biomarker Outcome Measures in Charcot-Marie-Tooth disease and Inclusion Body Myositis
  • Integration of pharmacodynamic biomarkers into a drug regulatory pipeline: Vamorolone/VBP15 in DMD
  • poster sessions  

Day 2

  • Big Data
  • Innovative bioinformatics solutions to identify genetic defects
  • Big data, large sequencing challenges, and the technology behind it
  • Neurology and Neurodegeneration Genomics England Clinical Interpretation Partnership (Neuro-GeCIP)
  • MRC Strategic plan
  • ohn Newsom-Davis Lecture The congenital myasthenic syndromes: better treatments through an understanding of disease mechanisms'

For more information visit the events page.