Getting a Diagnosis for Amyloidosis With ARC Founder Isabelle Lousada
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Lung Health
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Rare Diseases
Olympian William Flaherty’s Early Battle With a Rare Disease
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Why the Future Looks Bright for Geneticists and Their Patients
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Why It’s an Exciting Time in Rare Disease Drug Development
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How New Research and Treatments Can Help People With Hemophilia
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Innovative Program “Drafts” Researchers to Support People Impacted by Rare Diseases
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Bridging the Gap Between Pediatric and Adult Sickle Cell Care
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Gene Therapy Research Offers Transformative Possibilities for People Living With Hemophilia B
Hemophilia B is an ideal target for gene therapy because it’s caused by mutations in a single gene. Gene therapies under investigation aim to provide a working copy of the F9 gene.
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