During his most recent visit to Boston Children’s Hospital, 3-year-old Konrad Schienke resembles a tiny tornado, gleefully scampering around the room as he mugs for the camera and shouts, “Cheese!” Later, he smiles as a doctor gently felt his abdomen, giggling as if he was being tickled.
“It’s hard to believe what a sick little kid he has been,” says his father, Erich.
Yet, just a few years ago, this energetic boy resided in the neonatal intensive care unit at his local hospital in Pennsylvania, struggling with a diagnosis of short bowel syndrome. This rare but serious condition can occur when a child either loses or is born without enough small intestine, preventing the body from extracting the nutrients it needs to survive. Untreated, short bowel syndrome can lead to severe dehydration and malnutrition. …
Medical research is not a field for people who like instant gratification. There are long waits as cultures grow, proteins crystallize and cells divide. And when a discovery finally becomes something tangible, like a medication, it can still take years of testing and government approvals before a patient can benefit from it.
But what happens when a life is at stake and time is a luxury the patient simply cannot afford?
That is the question asked by NBC News’ Rock Center in a recent piece they did on Boston Children’s Hospital surgeon, Mark Puder, MD, PhD. Puder has help develop a potentially life saving drug called Omegaven, which he’s been using to help reverse fatal liver disease in infants. However, despite the drug’s effectiveness in Puder’s patients, the Food and Drug Administration has yet to approve its use nationwide.