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Not all of these new drugs came from big pharma as biotech companies like Regeneron (Eylea for wet acute macular degeneration), Vertex (Kalydeco for cystic fibrosis) and Incyte (Jakafi for bone marrow disease) had major drugs approved.
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Late last year, Jack and Lisa Nash, a Colorado couple, opted for the high-tech approach to avoid their new baby suffering from Fanconi anaemia, a rare inherited bone-marrow disease, which plagues their daughter.
ECONOMIST: Gene genies
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At his age he was almost 60 and after a decade of chronic bone marrow cancer, his disease was especially dangerous.
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Not even heavy chemotherapy and bone marrow transplants can vanquish the disease.
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The study tested whether myeloma patients who had stable disease following a bone marrow transplant did better if they received Revlimid or placebo.
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Few treatments, aside from bone marrow transplants, could actually stop the disease.
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Myelofibrosis is a disease that afflicts the bone marrow.
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It is a particular gamble if the child is conceived partly to provide a bone marrow graft for a brother or sister with a nasty genetic disease: not only might the new baby turn out be a poor match for transplantation, but might also have inherited the same disorder as his or her sibling.
ECONOMIST: Gene genies
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Of the millions of people with sickle-cell disease in the world, only some 300 have received a bone marrow transplant because of age (doctors won't perform the procedure on patients 16 years and older) and donor-matching problems.
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