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There are millions of people worldwide with rare diseases who are in need of accurate diagnoses and access to appropriate treatments. Many people share their stories and opinions with me. This blog is meant to give them voice so that together we can continue the global crusade for orphan drugs to treat rare diseases.

Access to the Book at Conference

"Orphan Drugs: A Global Crusade" was distributed at the 2018 meeting of the "Professional Patient Advocates in Life Sciences". After the meeting an attendee sent the following message to another person in the audience: 

Orphan Drug Tax Credits Save Lives

At the end of 2017 the U.S. Congress was deciding whether to eradicate orphan drug tax credits that alleviate 50% of the costs of "clinical research" when studying the safety and effectiveness of an orphan drug on human beings. Rare disease patients, charities, scientists and pharmaceutical companies objected to reducing or eliminating the tax credits. This article explains the value of orphan drug tax credits in spurring development of new treatments for rare diseases over more than 3 decades.

Radio programs examining the successes and controversies surrounding orphan drugs

The stories below are January 2017 articles and NPR radio programs about orphan drug issues that were written and published by Kaiser Health News (related to the Kaiser Family Foundation) and the National Public Radio (NPR) broadcast system. They are in-depth articles examining the successes and controversies surrounding orphan drugs and how they are affecting patients lives.