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RESEARCH TRIANGLE PARK - The nearly 30 million Americans who suffer from rare diseases have received some good news. The National Institutes of Health, the U.S.
A new national public/private consortium designed to boost the development of gene therapies also holds promise for North Carolina, as a major global leader in cell and gene therapies.
While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies.
The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to
A new, retrospective study of medical and insurance records indicates health care costs for people with a rare disease have been underestimated and are three to five times greater than the costs for people without a rare disease.