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.
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.