In collaboration with a pioneer PIGW-CDG family, we identified two over-the-counter nutraceuticals as actionable hits from a yeast screen. The combination treatment is working.
This is wonderful news about Hannes and it is so rewarding for me to be a part of this successful real-life patient story. It reminds me of my days as a postdoc funded by the PNW Mito Guild to work on Leigh syndrome using a worm model system, and I got the opportunity to meet patients impacted by this rare, debilitating disease. It's really a dream come true to contribute to Perlara's wonderful work.
This is wonderful news about Hannes and it is so rewarding for me to be a part of this successful real-life patient story. It reminds me of my days as a postdoc funded by the PNW Mito Guild to work on Leigh syndrome using a worm model system, and I got the opportunity to meet patients impacted by this rare, debilitating disease. It's really a dream come true to contribute to Perlara's wonderful work.