Subscribe to
this category


A total of 11 posts are filed under Innovation
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
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
Grace Science Foundation and Stanford University Create Biobank for Ultra-Rare NGLY1 Deficiency
Rare Daily Staff The Grace Science Foundation and a clinical research team from Stanford University have created an open source biobank of… Continue Reading