Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations. He did this in partnership with patients and patient advocates who had been unable to gain national attention until he lent his support. read more >
In an extraordinary signal of the progress that is being made to find treatments for some types of rare disorders, an advisory committee to the U.S. Food and Drug Administration (FDA) has recommended approval this week for not just one but two new drugs to treat homozygous familial hypercholesterolemia (HoFH).
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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