The Catalyst

Advancing the science for patients with rare diseases

Posted by Allyson Funk on February 29, 2016 at 9:00 AM

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 advances we saw in treatments last year and the hope we have for continued progress in the years ahead.

BannerYearforRareDisease.jpgRare diseases are those that affect fewer than 200,000 people in the United States, but with more than 7,000 different rare diseases known today, nearly 1 in 10 Americans is living with a rare disease. Eighty percent of rare diseases – including the one impacting my family – are genetic in origin, making the rarity of each disease often unknown and treatment options especially limited. Today, approved treatments are available for just 5 percent of rare diseases.

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Topics: Innovation, Rare Diseases, R&D, Rare Disease Day

Leading efforts to better understand amyloidosis diseases

Posted by Guest Contributor on February 26, 2016 at 9:00 AM

ARClarger.pngConversations 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 contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view. Views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today.

We’re pleased to host a guest blog from Isabelle Lousada, president and CEO of the Amyloidosis Research Consortium.

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Topics: Rare Diseases, R&D, Rare Disease Day

Why neglected tropical disease and rare disease researchers need each other

Posted by Guest Contributor on February 25, 2016 at 9:00 AM

Sanfilippo_image.jpgConversations 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 contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view. Views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today.

We’re pleased to host a guest blog from Jill Wood and Sean Ekins. Jill is the mother of Jonah, who has Sanfilippo Syndrome. She is the Founder of Jonah’s Just Begun, Co-Founder of Phoenix Nest, Inc. and a rare disease patient advocate. Her goal is to revolutionize efforts to bring treatments to rare disease patients. Sean is the CEO of Phoenix Nest, Inc.

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Topics: Rare Diseases, Rare Disease Day

Don’t lie down!

Posted by Guest Contributor on February 24, 2016 at 9:00 AM

 

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 contributors, including patients, stakeholders, innovators and others, to share their perspectives and point of view. Views represented here may not be those of PhRMA, though they are no less key to a healthy dialogue on issues in health care today.

We’re pleased to host a guest blog from Jamie Pires, a chronic myelogenous leukemia patient and advocate. Hear more about Jamie’s story here.


As children, we fantasize about the fairy tale: great success, fame and, of course, unlimited happiness. As we grow older, reality edges its way into our daily lives, and we start to understand that life is incredibly complicated. 

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Topics: Rare Diseases, Leukemia, Rare Disease Day

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