The Catalyst - A PhRMA Blog | Rare Diseases (3)

Leading efforts to better understand amyloidosis diseases

By Guest Contributor | February 26, 2016

Conversations and healthy debate about issues facing our industry and the health care system are critical to addressing some of today’s challenges and opportunities. The Catalyst welcomes guest... Read More

Why neglected tropical disease and rare disease researchers need each other

By Guest Contributor | February 25, 2016

Don’t lie down!

By Guest Contributor | February 24, 2016

Biopharmaceutical Companies Recognized as Meaningful Places to Work

By John Tunnell | May 22, 2015

Loretta was diagnosed with Stage 4 lung cancer 12 years ago - but thanks to medical breakthroughs and innovative medicines, she is living a happy, active life today. Matt was given a five percent... Read More

New Report Highlights a Decade of Innovation in Rare Diseases

By Stephanie Fischer | March 5, 2015

Despite Scientific Challenges, Biopharmaceutical Researchers Making Progress against Rare Disease While any given rare disease affects fewer than 200,000 people in the U.S., rare diseases... Read More

Week in Review: Keeping Patients In-The-Know

By Christian Clymer | February 27, 2015

Many patients are not aware of the harmful effects that non-adherence to needed medicines can have on their health, lifestyle and the economy. This week, we explored the ways in which medication... Read More

Working Together to Stop Ebola in its Tracks

By John Castellani | November 13, 2014

The fantastic news came yesterday that Dr. Craig Spencer, the last patient in the U.S. with Ebola, has been cured. While this development certainly inspires hope, there is still much to be done in... Read More

Empowering Patients to Be Active Participants in Clinical Trials

By Stephanie Fischer | October 2, 2014

One of the topics of discussion at the recent Rare Patient Advocacy Summit hosted by Global Genes was the role of patients in healthcare. A statement that clearly resonated with the audience,... Read More

Collaboration is What Will Help Drive New Successes for the Rare Disease Community

By Stephanie Fischer | March 26, 2014

While the number of patients with any individual rare disease is relatively small (less than 200,000 in the U.S., as defined by the Orphan Drug Act of 1983), they collectively affect 1 in 10... Read More

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... Read More

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