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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