Rare Diseases
Rare Diseases
Why the Future Looks Bright for Geneticists and Their Patients
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Rare Diseases
Why It’s an Exciting Time in Rare Disease Drug Development
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Rare Diseases
How New Research and Treatments Can Help People With Hemophilia
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Rare Diseases
Innovative Program “Drafts” Researchers to Support People Impacted by Rare Diseases
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Sponsored
Bridging the Gap Between Pediatric and Adult Sickle Cell Care
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Sponsored
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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Rare Diseases
Building Health Equity for Rare Diseases
Rare Diseases
The Uncommon Cause of Rare Disease
Rare Diseases
How This Fitness Coach and Influencer Took on Cystic Fibrosis and Won
Neurological Disorders
The Economic Impact of Rare Disease
Black Health
The Barriers Black Families Face When Confronting Rare Disease
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Rare Diseases
Why Genetic Testing Is the Key to Earlier Rare Disease Diagnoses
For those with a primary immunodeficiency, one of more than 450 rare conditions that affect the immune system, genetic testing holds the key to early diagnosis and appropriate treatment.
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Rare Diseases
How the Littlest Things Can Make the Biggest Differences in Rare Disease
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Rare Diseases
Accelerated Approval Delivers Hope for Rare Disease Patients
Rare Diseases
Compassion in Medicine: Why Caring Matters
Rare Diseases
A Potential Breakthrough Treatment for Arginase 1 Deficiency Is on Its Way
Rare Diseases
Prenatal Genetic Tests Offer New Noninvasive Options