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Glyn's Gift

The beginnings of CDKL5
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Natural History Study

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iPS Disease Modeling and Drug Development in CDKL5

Dr. Muotri will attend the IFCR Science Symposium to be held in the Chicago, IL, area in June. He has ... Learn More

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Roadmap to a Cure


 

Estimated Time Lines and Costs

Mouse Models   completed
  • Need 3-4 mouse models with reproducibility
  • 1-1.5 yr/ mouse
  • $100,000/ mouse
  • Characterization: $50,000/mouse
iPS Cells  completed
  • Need 3-4 cell lines with reproducibility
  • Correlation to Mouse lines helpful
  • $100,000 to $300,000
  • 6-8 months per line
Protein Target Identification  in progress
  • Proteomics $50,000
  • Mass spectrometry $30,000
  • 1-2 years
Drug Discovery, Screening and Development  in progress
  • $2.5 million
  • 2-3 years
Phase I (Drug) Clinical Trial
  • $250,000
  • 2 years
Phase II (Pilot) Clinical Trial
  • $1.8 million
  • 1.5 years
Phase III Clinical Trial
  • $8 million
  • 3 years
Natural History Study  enrolling now 
 
CDKL5 Clinical consortium  in progress 
  • ongoing
  • $150,000 per year
DATABASE  in progress
  • Ongoing
  • Critical for determining Therapeutic Endpoints for Trials
  • $75,000

 

Roadmap to a Cure

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