Belangrijkste was ik toch bijna vergeten, hoop nu dat MEER mensen/beleggers nu begrijpen wat Pharmings Ruconest kan betekenen en is dit de aanzet hiervoor (Covid 19)
There are multiple other protein deficiency disorders that Pharming could apply its technology to, Herrmann added, notably Pompe, Fabry and Gaucher's diseases — all of which are rare diseases known as lysosomal storage disorders, and represent a global market opportunity worth some $5.6 billion in 2017.
People with HAE are unable to make C1 esterase inhibitor, which is the handbrake of 90% of human inflammatory processes, including most of those that involve fatal cytokine storms, said CFO Robin Wright. As a consequence, sepsis, kidney injury and meningitis are all potential further indications for Ruconest.
"In a cytokine storm situation — such as is caused by pre-eclampsia, contrast-induced nephropathy, kidney injury, meningitis, sepsis — the pro-inflammatory molecules just swamp any production of C1 that is meaningful," said Wright. "So you have to supply if you want to treat it. The idea is if you supply lots of C1 early on, you can slow that storm down, so that it doesn't cause so much damage and ideally doesn't kill the patient. All of these indications are linked by that fundamental concept."