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Group 5 Pulmonary Hypertension: The Orphan's Orphan Disease.
Academic Article
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Therapeutic options for variant renal cancer: a true orphan disease.
Academic Article
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Genetic regulatory variation in populations informs transcriptome analysis in rare disease.
Academic Article
Why?
The Challenge of Defining Treatment Standards for a Rare Disease: Malignant Peritoneal Mesothelioma.
Academic Article
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The genetic key to a rare disease and its impact on orthopedics.
Academic Article
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Studying a Rare Disease Using Multi-Institutional Research Collaborations vs Big Data: Where Lies the Truth?
Academic Article
Why?
An Orphan Disease No More: Additional Treatment Options for Cardiac Amyloidosis.
Academic Article
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Using Registries to Understand Clinical Practice: A Lesson for Rare Disease.
Academic Article
Why?
Rare Diseases
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Molecular genetic testing for ultra rare diseases: models for translation from the research laboratory to the CLIA-certified diagnostic laboratory.
Academic Article
Why?
Naming and understanding rare diseases: International Classification of Diseases coding and the epidemiologic designations of idiopathic pulmonary arterial hypertension.
Academic Article
Why?
Pulmonary arterial hypertension: future directions: report of a National Heart, Lung and Blood Institute/Office of Rare Diseases workshop.
Academic Article
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Adolescents and young adults with a "rare" cancer: getting past semantics to optimal care for patients with germ cell tumors.
Academic Article
Why?
Clinical trial strategies for rare neurodevelopmental disorders: challenges and opportunities.
Academic Article
Why?
Management of pediatric myxopapillary ependymoma: the role of adjuvant radiation.
Academic Article
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