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Science

A total of 33 posts are filed under Science
Addressing the Challenges of R&D for Rare Cancer Therapies
Rare cancers, like other rare diseases, face the challenges of mustering research attention, access to patients and biological materials,… Continue Reading
A Victory for One
In 1886, the physician Jonathan Hutchinson examined a six-year-old patient who lacked hair and had the withered skin of an old man. Later,… Continue Reading
NEJM Publishes Promising Bluebird Bio Gene Therapy Data in Beta Thalassemia
Rare Daily Staff Interim data published in the New England Journal of Medicine from two separate studies of Bluebird Bio’s LentiGlobin,… Continue Reading
FDA Clears for Commercial Use Exemplar Genetics’ Animal Model for Rare Neurodegenerative Disease
Rare Daily Staff The U.S. Food and Drug Administration cleared Intrexon subsidiary Exemplar Genetics’ ExeGen ATM MiniSwine model for… Continue Reading
Erectile Dysfunction Drugs Have Potential to Treat Rare Cancers
Rare Daily Staff The class of erectile dysfunction drugs that include Viagra, Cialis, and Levitra, known as PDE5 inhibitors could be… Continue Reading
NIH Researchers Establish Genetic Cause of Rare Bone Disorder
Rare Daily Staff Researchers at the National Institutes of Health uncovered a genetic basis of melorheostosis, a condition also known as… Continue Reading
Researchers Declare Breakthrough in Rare Brain Disease in Kids
Rare Daily Staff Researchers from Université de Montréal and the CHU Sainte-Justine and Université de Montréal Hospital say they have… Continue Reading
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