Tag Archives: orphan diseases

Missed Opportunities in Rare Disease

By Ben Comer | Published: July 20, 2012
At CBI’s 7th Annual Rare Disease and Orphan Drug Leadership Congress, speakers and attendees identified areas where pharma can improve the way it approaches rare disease and treatment.
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Rare Disease Day: Spotlight on Solidarity

By Jennifer Ringler | Published: February 28, 2012
Patients, policymakers and pharmaceutical companies are uniting around rare diseases on the rarest of calendar days, presaging a leap forward in health outcomes and business opportunities. Aligning humanistic objectives with business incentives is often the sine qua non of effective community activism. This year’s Rare Disease Day – hosted in the US by the National [...]
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Creating Hope For Orphan Diseases

By Jennifer Ringler | Published: June 8, 2011
The Creating Hope Act of 2011, along with advancements in science and a shift in focus for Big Pharma, means that rare and pediatric orphan diseases may finally get the attention they deserve. “As a society, as we’re having this big debate around healthcare, everyone agrees that there’s a certain baseline of people that we [...]
Posted in FDA, Gene therapy, Regulatory, healthcare | Also tagged , , | 1 Comment