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July 9, 2014

TOPIC: Advocacy, Patients & Members

Knowing More About Noonan

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Katie Thortenson of Chicago is a proud mother of two healthy sons: her eldest, Jake, is 6, and her youngest, Drew, is 2, but often mistaken to be younger. When people ask why he’s so small, Katie explains… Read More

June 23, 2014

TOPIC: Patients & Members

Living With Essential Thrombocythemia (ET)

Posted by Mary Dunkle

In 1992, Antje Hjerpe was diagnosed with a rare blood disease known as “essential thrombocythemia” or ET. A review of previous lab records showed that she had  been living with this disease for at least six years prior to diagnosis. (more…)

June 17, 2014

TOPIC: Advocacy, Featured News, Industry, Medical, Patients & Members, Research

NORD Recommendations for Advancing Drug Discovery, Development, and Delivery

Posted by Mary Dunkle

On behalf of the millions of Americans who have diseases with little or no treatment, the National Organization for Rare Disorders (NORD) has submitted a seven-step plan to the 21st Century Cures Initiative outlining ways to improve the discovery, development, and delivery of medical treatments in the U.S. Read More

June 4, 2014

TOPIC: Medical, Patients & Members, Research

Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes

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Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of rare disease patients have found their lives bettered by biologics and we are collectively grateful to the innovators and companies that have made this possible. Read More