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topic Health • rss.nytimes.com • 8 hours ago

‘Too Rare to Care’? A New Center for Rare Diseases Hopes to Change That

‘Too Rare to Care’? A New Center for Rare Diseases Hopes to Change That
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Why This Matters

Medical breakthroughs like this offer hope to patients worldwide and represent years of dedicated research improving lives.
A new nonprofit wants to streamline gene therapy for diseases often avoided by pharmaceutical companies — making treatment more like a routine procedure than a bespoke drug.
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