Modulator drugs interact with mutant CFTR and force abnormally-folded proteins into a more functional structure. But these modulators are mutation-specific, and Skach estimates that 7% of cystic ...

The neuronal aggregates that are observed in HD cases are not only composed of mutant huntingtin but also of other proteins, including normal huntingtin 11. In this regard, it is possible that ...

Join Peter Grootenhuis of Vertex Pharmaceuticals as he delivers a brief overview of the drug discovery approaches and challenges to identify small molecules CFTR modulators. These compounds improve ...