Could a form of sugar treat deadly Niemann-Pick disease? | fox4kc.com
It wouldnt be simple. Cyclodextrin wasnt a medicine; the Hempels had to order it from a bulk supplier in Florida and have it mixed and sterilized at a compounding pharmacy. Whats more, you cant just go into a hospital and inject something new into a patient. The Food and Drug Administration wanted proof it was safe. That might have been an insurmountable hurdle. Safety studies, even in animals, typically take years and cost millions of dollars. But this case was different.
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Otherwise, how would anyone know if the treatment worked? We need to get an answer, says Dr. Marc Patterson, a leading NPC expert at the Mayo Clinic in Minnesota. If you do it at NIH, its controlled, and its likely the data will help show whether or not it works. If you do it in your own child, its hard unless you have a treatment cost of plastic surgery operation that really reverses the symptoms. Dr. Forbes Porter, the scientist running the NIH study, is blunt.
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Cleveland Clinic does its 2nd face transplant – Quincy Herald-Whig | Illinois & Missouri News, Sports
In a statement released by the hospital Tuesday, the man said he is “grateful beyond words to the donor and his family for their amazing gift.” The patient became a candidate for a face transplant after many tries to reconstruct his face failed to improve his quality of life, the hospital said in a statement. The man had trouble breathing and speaking, and the transplant offered the chance to save the limited sight in his sole remaining eye. Doctors transplanted about two-thirds of the scalp, the forehead, upper and lower eyelids, eye sockets, nose, upper cheeks, upper jaw, upper teeth, salivary glands and nerves, muscles and skin. The patient is recovering well, breathing without a tube and is expected to be able to eat soon, the hospital said. He will need medicines for the rest of his life to prevent rejection of the new face. The Cleveland Clinic did the nation’s first face transplant, in December 2008, led by Dr.
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