Orphan Drug

Partnerships between patient groups, industry, government and academia are accelerating the research and development of new treatments for rare diseases, many of which have no effective therapies. As a result of the Food and Drug Administration’s (FDA) Orphan Drug Designation, eligible companies receive incentives for product development such as tax credits for qualified clinical testing and new drugs are reaching patients sooner. Since 1983, more than 400 drugs and biologic products for rare diseases have been developed, far outpacing the number of products that were available some 40 years ago. Amyotrophic lateral sclerosis (ALS) is among the nearly 7,000 rare diseases where very few...

Sidebar Quote

You can change the image of things to come. But you can’t do it sitting on your hands … The science community should reach out to Congress and build bridges.
The Honorable John E. Porter