Tag Archives: rare diseases

Mito What???

Liz-Kennerly-115x76

Mitochondrial Disease: The Most Common Disease You’ve Probably Never Heard of… About 20 years ago, mitochondrial disease was virtually unheard of.  Just 10 years ago, it was considered rare. Today, according to NORD, a disease is considered rare when less than 200,000 people are affected.  Mitochondrial disease is not a single disease, but rather an entire umbrella of rare diseases.  For example, Alpers disease and Barth syndrome are very different and rare forms of mitochondrial Read More >

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Rare Disease Day 2014: Joining Together for Better Care

Hans Schikan of Prosensa

There are more than 6,000 different rare diseases affecting over 60 million people across Europe and North America, alone, and millions more throughout the world. Most of these diseases are genetic, serious, chronic and debilitating. Each disease is different, but they affect people in similar ways. Joining together can help patients and their families find common solutions for care and remind them they are not alone. As the world marks Rare Disease Day on February Read More >

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The Ethics of the Market Access Debate

Rogerio Vivaldi

At the core of the market access conversation lies the question of value. How much do we value two years added to the end of an 80-year-old’s life? How do we put a dollar amount on a medicine that prevents a rare disease patient, who may only live to age 20 regardless of receiving treatment, from losing her ability to walk? How can we define metrics to measure the cost of a child with a Read More >

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A Bright Future for Orphan Disease Indications

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In celebration of National Rare Disease Day BIO’s Industry Analysis team took a look at recently-approved drugs in the BioMedTracker database with an orphan indication designation. Our research found that 2012 saw the greatest number of approvals of therapies for orphan indications going back all the way to 2000 (This count includes previously approved drugs with new, orphan indication approvals).   Overall, 24 therapies were approved in orphan indications last year, including drugs for Anthrax, Gaucher’s Read More >

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Navigating the Rare Disease World

Susan Ruediger

The international orphan drug marketplace is expected to grow some 30 percent by 2014, fueled by the entrance of big pharmaceutical companies and U.S. legislation favoring development and coverage. This growth promises to improve the health of patients with debilitating and life-threatening disorders. Hoping to further the rare disease dialogue and relationship building opportunities, the 2013 Orphan Disease Forum will provide unique perspective on the orphan drug marketplace, regulatory considerations and much more. Operating within Read More >

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