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Tackling Rare and Orphan Diseases

In the US, rare diseases are defined as those that affect fewer than 200,000 people. But there are more than 6,800 of these conditions, and worldwide they afflict hundreds of millions of people, and only about 250 treatments are available.

Editors’ Note: August 4, 2017
Legitimate objections have been raised about the independence and integrity of the commentaries that Henry Miller has written for Project Syndicate and other outlets, in particular that Monsanto, rather than Miller, drafted some of them. Readers should be aware of this potential conflict of interest, which, had it been known at the time Miller’s commentaries were accepted, would have constituted grounds for rejecting them.

STANFORD – Rare diseases are commonly neglected, simply because they are, well, rare. In the aggregate, however, they affect huge numbers of people. In the United States, rare diseases are defined as those that affect fewer than 200,000 people. But there are more than 6,800 of these conditions, and worldwide they afflict hundreds of millions of people. And yet only about 250 treatments are available for these diseases.

One reason for this is that small numbers of patients make studying illnesses and testing drugs difficult. As a result, medical professionals often know little about the symptoms and biology of these diseases.

Earlier this year, I participated in a Rare Disease Day program in California. The topic was an obscure but large collection (more than 70) of diseases caused by a specific type of genetic error – one that takes place in the machinery of cells that chemically attaches sugars to proteins and lipids.

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