Advancing the science for patients with rare diseases

When you have a loved one with a rare disease, the importance of persevering to find new, innovative treatments takes on new meaning. This year on Rare Disease Day, I am excited to celebrate the...
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Leading efforts to better understand amyloidosis diseases

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...
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Why neglected tropical disease and rare disease researchers need each other

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...
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Don’t lie down!

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...
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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...
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New Report Highlights a Decade of Innovation in Rare Diseases

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...
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Week in Review: Keeping Patients In-The-Know

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...
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Working Together to Stop Ebola in its Tracks

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...
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Empowering Patients to Be Active Participants in Clinical Trials

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

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 Americans
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