John Crowley describes the difficulties of getting his children with Pompe disease into clinical trials for treatment and ultimately saving their lives.
A father recalls the desperate struggle to get his children treated for Pompe Disease, including resigning from his job and reaching out to doctors directly, after a clinical study was stalled due to executive conflicts of interest.
A man shares his emotional experience of receiving a card from a donor family whose son had died and donated his organs which saved the man's life. He waited till Christmas Eve to share the card with his loved ones and it was a special moment for everyone.
Developing drugs requires rigorous testing of safety and effectiveness in animal models, and investing in plant development and building within a cleanroom can increase production efficiency.