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7,000+ rare diseases remain untreatable. The genetic revolution and federal research funding offers hope for cures, but vaccine hesitancy and a lack of newborn screening pose hurdles

In these days of the never-ending pandemic, other health problems continue to take a backseat. That’s especially true for the 7,000 or so rare

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The New Opportunity to Help Rare Disease Patients

In 2020, 46.9% of all novel drugs approved by the FDA were for rare diseases, as compared to 23.5% in 2012. Investment is expected to continue trending upward, with estimates suggesting that global spending on rare disease therapies will reach $260 billion by the end of 2025.

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The Economic Burden of Rare Disease - The 2023 NEXT Report

The Economic Burden of Rare Disease - The 2023 NEXT Report
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Why the underestimated economic burden of rare diseases could be costing the U.S. trillions of dollars

While rare diseases impact a small population, the cost to society is staggering. It may be in the range of $7.2-$8.6 trillion per year.

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Examining the Legislative Landscape for Rare Disease Drug Development

Examining the Legislative Landscape for Rare Disease Drug Development
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