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More than 10,000 known rare diseases affect over 30 million people in the United States. However, treatment options have been restricted due to small patient populations and limited commercial viability. Given…
Read guide →Karen Weintraub Contributor GEN Gene Therapy Allows Hemophilia Patients to Give Up Medication, Avoid Bleeds; but More Improvements Are Needed A new gene therapy trial in hemophilia B patients is the most succe…
Read guide →New Rochelle, NY, December 12, 2011—A recent article in the New England Journal of Medicine showed that Factor IX gene therapy in patients with hemophilia B was able to convert severe hemophilia to moderate or…
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