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WORLD’S MOST COMMON, LIFE-THREATENING GENETIC DISEASE


Discovery Provides Hope for 600,000 Americans Living with PKD


(Kansas City) March 20, 2006 — Researchers have found the drug rapamycin greatly reduces the size of enlarged kidneys in patients with polycystic kidney disease (PKD).


Rapamycin, also called sirolimus, is an immunosuppressant. The drug is prescribed to help prevent rejection of a new, transplanted kidney. Researchers tested rapamycin in mice and also in a small group of transplant patients. They found the patients’ polycystic kidneys shrank by 25 percent over two years.


PKD affects more people than Down syndrome, cystic fibrosis, muscular dystrophy, sickle cell anemia and hemophilia combined. For the 600,000 Americans suffering from polycystic kidney disease, (PKD) fluid-filled cysts grow throughout the kidneys. Over time, these cysts get bigger and multiply, often leading to kidney failure. There is no treatment or cure for PKD, but there is hope.


This study comes on the heels of another exciting research announcement. In February, the Food and Drug Administration granted “Fast Track” designation for Tolvaptan, a potential treatment for PKD.


“These advancements bring hope to the 12.5 million people world-wide living with Polycystic Kidney Disease (PKD),” said PKD Foundation President and CEO Dan Larson.


March is National Kidney Month, and PKD patients across the country are available for interviews on the disease and potential treatments. For more information on any of these topics, visit www.pkdcure.org or call 1-800-PKD-CURE.

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