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gene therapy workABC News
•84% Informative
About 350 million people worldwide suffer from rare diseases, most of which are genetic.
Each of the 7,000 individual disorders affects perhaps a few in a million people or less.
There’s little commercial incentive to develop or bring to market these one -time therapies.
Scientists say this dynamic threatens to thwart progress in the nascent gene therapy field.
Most genetic conditions are rare — each affecting fewer than 200,000 people in the U.S. at any given time.
Research hasn't made it past early stages for many of them.
Financial disincentives plague the process, from drug discovery to development, scientists say.
Last year , the Telethon Foundation in Italy took on responsibility of producing and distributing Strimvelis .
The charity announced it was selected for a pilot program of the European Medicines Agency that could help guide its gene therapy.
Scientists say these efforts don’t negate the larger financial quandary surrounding therapies for rare diseases.
VR Score
89
Informative language
89
Neutral language
75
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English
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55
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Attention-grabbing headline
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