Rare diseases

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A Rare Disease Halts a Navy Pilot’s Dreams, But Gives Him a…
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Blue Platelets
Working to Develop a More Safe and Convenient Treatment…
While many people have heard of the blood disorder hemophilia,…
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Get Science Podcast: An Urgent Quest to Bring 'True…
Developing treatments for rare
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Breaking the Rules of Biology to Beat Rare Genetic Diseases
Like a skipping record, our
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From Flask to Vat: The Challenges of Scaling Up Gene Therapy
Over the past two decades,
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Hope in Translation: Bringing Gene Therapy to the Clinic
In 1987, scientists first uncovered the gene that is the target…
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Bespoke Is Often Better: How Scientists Are Customizing…
When it comes to products from suits to kitchen cabinetry,…
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How Does It Feel to Have Sickle-Cell Disease?: When…
Molecular biologists certainly have the insider’s view of a…
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In Search of a Better Biomarker: Why Studying Liver Enzymes…
If you’ve ever had a liver function test as part of an annual…
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