casgevy
3 stories tagged "casgevy"
Sickle Cell Finally Has a Cure. It Costs $2.2 Million and Has Reached 64 People.
Casgevy has kept Victoria Gray crisis-free for seven years. Getting it still means wiping out a patient's bone marrow with a chemo tied to infertility and malignancy warnings and paying about $3 million all in, and two years after approval only 64 people have been infused.
Drugs & FDAThe FDA Cleared a CRISPR Cure for Two-Year-Olds Whose Own Trial Enrolled No One That Young
Casgevy's pediatric trials enrolled 11 children aged five and up, yet the FDA extended the $2.2 million therapy down to age two, where the conditioning chemo can permanently sterilize a child too young to bank eggs or sperm.
Drugs & FDAThe FDA Cleared a Sickle-Cell Cure for Two-Year-Olds in 53 Days. It Starts With Chemo That Can Sterilize Them.
Casgevy is a real advance against a brutal disease. It is also a $2.2 million therapy that requires sterilizing chemotherapy, saw a child die in trials, and reached toddlers on a 53-day review Makary's FDA hands out as a favor.