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A total of 14 posts are filed under Innovation
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
Novel Cell Therapy Restores Dystrophin in Mouse Model of DMD
Rare Daily Staff Researchers report that they significantly improved muscle function in a mouse model of the rare and fatal condition… Continue Reading
NIH to Launch Research Program to Develop Tools for Genome Editing
Rare Daily Staff The National Institutes of Health said it is launching an effort to remove barriers that slow the adoption of genome… Continue Reading
Failed COPD Drug Shows Promise as Treatment for Rare Bone Disease
Rare Daily Staff A drug Roche had unsuccessfully sought to develop as a treatment for chronic obstructive pulmonary disease has shown… Continue Reading
Transgenic Stem Cell Therapy Restores Skin of Boy with Rare Genetic Condition
Rare Daily Staff A seven-year-old boy in Germany who lost 80 percent of his skin to a potentially lethal rare genetic condition that causes… Continue Reading
University of Bonn Institutes Embrace FDNA’s Facial Analysis to Improve Diagnosis of Rare Diseases
Rare Daily Staff The Institute for Genome Statistics and Bioinformatics and the Institute of Human Genetics of the University Hospital of… Continue Reading
Sarepta Therapeutics Enters Collaboration with Duke University to Develop Gene Editing to Treat DMD
Rare Daily Staff Sarepta Therapeutics said it signed a research collaboration agreement with Duke University that grants the company an… Continue Reading

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