New Drugs For Rare And Deadly Diseases Now In Testing Phase

WASHINGTON (January 16, 2013)—Drug makers are doing much more research on rare, often-deadly diseases with few or no treatment options, and that’s good news for the nearly 30 million Americans with such an illness.

The drug makers are running patient tests of more than 5,400 potential new medicines, many for multiple conditions; nearly 1,800 research projects are for rare diseases and hundreds more are for disorders with no new treatments in many years, according to a study that will be released Thursday by the trade group Pharmaceutical Research and Manufacturers of America.

The study found that thousands of those experimental drugs could be the first in a new medicine class with a unique target or effect.

Nearly 600 of them use new technologies, from gene therapy to cloned antibodies, to precisely target the disease site while others are for tiny subgroups of patients with genetic variations.


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