“Developing a mutant protein-stabilizing platform based on a small molecule for orphan diseases”

In Featured on App, Orphan Drug Congress by Freya Smale

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Zohar Argov of BioBlast Pharma  joined us for World Orphan Drug Congress USA 2015 to present about ‘ Developing a mutant protein-stabilizing platform based on a small molecule for orphan diseases’ 
Download the presentation now to learn about:

  • Trehalose as a known protein stabilizer and autophagy enhancer
  • Proof of Concept: Trehalose in OPMD
  • Current human studies of Cabaletta

…and much more!

Get your copy here !

 To learn more about developing a mutant protein-stabilizing platform , attend World Orphan Drug Congress USA 2016 September 2-3  in Washington, DC.