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Adult cystic fibrosis patients account for like a third of all lung transplants in the US and children with CF account for like half of all pediatric lung trans
by DoreenMichele 4y ago
Adult cystic fibrosis patients account for like a third of all lung transplants in the US and children with CF account for like half of all pediatric lung transplants. Lung transplants cost like a million dollars, can lead to a gruesome death if rejection sets in and require lifelong drugs.
The latest, greatest drugs for CF are extremely expensive (around $250k annually last I checked), don't work for everyone and must be taken forever as well.
Standard treatments for CF are extremely expensive and the condition is extremely debilitating.
It's a good candidate for new gene editing therapies in part because there is a simple, identified issue: a defective cell channel. Some genetic disorders are more complicated than that.