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JAMA. 1991;265(23):3128-3130. doi: 10.1001/jama.1991.03460230078019

Hematology

  1. Bruce Furie, MD;
  2. Kenneth B. Miller, MD
  1. New England Medical Center and Tufts University School of Medicine, Boston, Mass

Since this article does not have an abstract, we have provided the first 150 words of the full text.

Excerpt

After several decades of fermentation, the explosion of new knowledge in the life sciences is yielding practical benefit to all; the investment of public funds in basic biomedical research over the last two decades has given birth to new understanding of disease processes, new diagnostic methods, and a new generation of therapeutics. Last year was pivotal in hematology with regard to the clinical application of these technologies. Advances in protein biochemistry, molecular biology, and cellular biology have opened new vistas for the study and treatment of many hematologic diseases.

The diagnosis and treatment of hemophilia has been a prototype for the study and treatment of other genetic diseases. Although the severity of the hemophilia phenotype has been known to be variable in different families, only now do we appreciate the biological analogue of Murphy's Law: whatever can go wrong will. Hemophilia is a heterogeneous disease characterized by the deficiency of

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