How To Turn Stalled Research Into Treatments For Ultra-Rare Diseases w/ Stephanie Manton
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A therapy can be scientifically obvious and still never reach a patient. That contradiction is the centre of our conversation with Stephanie, CEO of WinQure Medicine, where we pull apart what really breaks when you try to develop drugs for ultra-rare disease, including “nano-rare” patients who may be one in a million.
We start with the lived reality: parents and carers who become accidental biotech founders, learning toxicology, regulatory approval, clinical operations, and manufacturing because the standard pharmaceutical machine is tuned for large markets. Stephanie shares…
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