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NORD on the Need for More Collaboration to Drive Progress for Rare Disease Patients

By Stephanie Fischer  |    February 17, 2014
Many biopharmaceutical companies are actively engaged in the research and development of therapies to treat rare disease, despite challenges such as much smaller patient populations than more common...   Read More

#RarePOV Conversation Generates Strong Enthusiasm for Rare Disease Day on February 28th

By Stephanie Fischer  |    February 12, 2014
In last week’s #RarePOV tweetchat, leading rare disease advocacy organizations and individual patient advocates came together to share resources and plans for the upcoming international Rare Disease...   Read More

Addressing Challenges in Rare Disease Drug Development - Together

By Stephanie Fischer  |    January 27, 2014
Dr. Richard Moscicki, Deputy Center Director for Science Operations at the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), noted in his remarks  at the FDA public...   Read More

Rare Disease Community Loses a Champion

By Stephanie Fischer  |    January 15, 2014
The rare disease community lost a champion last week when Sam Berns passed away at age 17 due to complications from progeria, an extremely rare genetic disease which causes accelerated aging.  Last...   Read More

Rare Disease Day Should Last All Year

By PhRMA Staff  |    March 9, 2011
Check out the Huffington Post blog by PhRMA President & CEO John Castellani and Gregg LaPointe, CEO of Sigma-Tau Pharmaceuticals, Inc. They look at some of the unique research hurdles in the search...   Read More

Setting the Story Straight on Rare Diseases

By PhRMA Staff  |    March 4, 2011
A column in The Boston Globe today tells the exciting story about a local company that is reporting some very positive results from its development program for a medicine to treat cystic fibrosis...   Read More

Rare But Equal

By PhRMA Staff  |    March 1, 2011
The theme to Rare Disease Day 2011 was "Rare But Equal." I glossed over that at first when I saw it, but the more I thought about it, the more chilling it is.   Read More

Because One Day Isn't Enough

By PhRMA Staff  |    February 28, 2011
Today is Rare Disease Day, a day meant to raise awareness of the 25 million Americans living with diseases that are often little-known and often, unfortunately, undertreated. In conjunction with Rare...   Read More

Searching for Success in ALS

By PhRMA Staff  |    February 10, 2011
Yesterday, our friends at ALS Advocacy Tweeted about the Chart Pack: "Need some #ALS success charts in the next edition!" They can rest assured that America's biopharmaceutical research companies are...   Read More

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