Tag Archives: orphan

BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

BIO Investor Forum

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases. Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring Read More >

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New Rare Disease Miniseries

roulette

Calling all biopharma researchers! Morning show “The Balancing Act” is producing a miniseries called “Roulette Wheel of Rare and Genetic Diseases,” and they are looking for pharmaceutical partners to help educate their audience. The series is working with the Global Genes Project to help educate patients and their families about new research, clinical trials, and government policies that affect those with a rare disease. Patient groups are already excited about this opportunity to spread awareness- take Read More >

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2012 Orphan Disease Forum: Addressing Today’s Opportunities and Challenges

disease

If all of the people with rare disease lived in one country, it would be the world’s third most populous country! Through dedicated companies such as the National Organization for Rare Disorders (NORD) and Centric Health Resources, the pressing issue of orphan diseases and its patients are being addressed. A rare disease is defined by any disease affecting fewer than 200,000 Americans. There are nearly 7,000 such diseases affecting over 350 million people worldwide.  Furthermore, Read More >

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BIO Europe Spring 2012 – Opening Remarks

For Monday’s Opening Remarks at BIO Europe Spring 2012, I covered the following topics: Public market performance in the US vs other sectors Private funding environment. Drug pipeline deal volume. For a copy of the slides, they can be found here.

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