A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.
A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.
Some ghoul company will charge about $2M for this therapy, which a few parents can pay, but most cannot and hence, fuck those kids. That is the American way.
This has been happening with SMA disease for years. Novartis gives away doses by lottery. You win, yay, you lose, your baby dies.
Vasant Narassimhan sleeps very well in a mansion in Boston. He makes $13,000,000 a year.