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Rare Disease

A total of 13 posts are filed under Rare Disease
Why Patient-Centred Outcomes Are Critical to Rare Disease Drug Trials
  Despite growing acceptance that patients have the clearest view of the health outcomes that matter, drugs to treat rare diseases often… 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
Learning from Rare Diseases
Matt Wilsey has liked to make the case that one justification for investing in rare disease research is that through it, we can unlock an… Continue Reading
Gene Editing Technology in Mice Shows Promises as Approach for DMD
Rare Daily Staff Scientists at the University of California, Berkeley have developed a way to deliver gene-editing technology inside cells,… Continue Reading
Study Suggests Existing Drugs Could Treat Rare Bone Cancer
Rare Daily Staff Some patients with chordoma, a rare bone cancer, could benefit from a class of approved drugs known as P13K inhibitors,… Continue Reading
UMass Medical School Gets $10 Million Gift from Li Weibo to Create Rare Diseases Research Institute
Rare Daily Staff The UMass Medical School announced the establishment of the Li Weibo Institute for Rare Diseases Research, an effort to… Continue Reading