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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

Ricki Lewis | 
There are an estimated 7,000 known rare diseases affecting 30 million people; for 95% of them, there are no treatment ...
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Unconventional drug ‘lottery’ will hand out 50 doses of Zolgensma, world’s most expensive gene therapy

Andrew Joseph, Ed Silverman | 
The lottery that began [recently] was not about money, or about choosing a school, or about obtaining a visa. It ...
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Viewpoint: Outrage over $2 million price tag for new gene therapy is perplexing and disappointing

Nathan Yates | 
As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and ...
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