Tag Archives: NORD

NORD President: Congress must act now to help 15 million sick children


Writing in The Hill, National Organization for Rare Disorders (NORD) President and CEO Peter Saltonstall called upon Congress to act swiftly to maintain an important program encouraging research into treatments for rare pediatric diseases. The Rare Pediatric Priority Review Voucher (PRV) Program is set to expire at the end of the month, unless Congress reauthorizes it. Saltonstall writes: Developing therapies for people with rare diseases is incredibly difficult. Rare diseases are scientifically and medically complex, Read More >

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The Washington Post Unfairly Attacks The Orphan Drug Act


The Orphan Drug Act has been a tremendous success and addressed a market failure by providing incentives to bring new treatments to those suffering with rare diseases. That’s why I was deeply troubled by Friday’s unfair article in The Washington Post targeting the Orphan Drug Act through selective and unsubstantiated attacks that seem designed to undermine this bipartisan and hugely successful regime that has helped so many patients with rare diseases live longer and healthier Read More >

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Orphan Drug Tax Credit is Crucial to Rare Disease R&D


The term “rare disease” may be misleading: to be “rare” a disease must effect fewer than 200,000 people in the U.S., but there are over 7,000 rare diseases that have been identified. Taken together, that means that nearly 30 million Americans suffer from a rare disease or condition.  While innovative therapies have been developed for a variety of these diseases—including Sickle Cell Anemia, Gaucher Disease, and Huntington Disease—too many patients still lack treatment options. Developing Read More >

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Complexities of Rare Diseases Take the Spotlight at BIO


Global Genes Founder & CEO Nicole Boice opened the BIO 2015 Orphan & Rare Disease Track by addressing a packed session with a simple, powerful statement: “We’re all working to eliminate the challenges of rare diseases, but patients and their advocates aren’t always equipped to fight.” Organizations like Global Genes help families affected by rare disease by connecting them with much needed tools and resources. By better understanding the definition of a “rare” disease, industry Read More >

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Mito What???


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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