Subscribe to
this category


A total of 488 posts are filed under Innovation
Ultragenyx and Kyowa Kirin Win FDA Approval for Crysvita to Treat XLH
Rare Daily Staff The U.S. Food and Drug Administration granted approval to Crysvita, Ultragenyx and Kyowa Kirin International’s treatment… Continue Reading
Lonza Opens World’s Largest Dedicated Cell-and-Gene-Therapy Manufacturing Facility
Rare Daily Staff The biopharmaceutical manufacturing giant Lonza said it has opened the world’s largest dedicated cell-and-gene-therapy… Continue Reading
Erectile Dysfunction Drugs Have Potential to Treat Rare Cancers
Rare Daily Staff The class of erectile dysfunction drugs that include Viagra, Cialis, and Levitra, known as PDE5 inhibitors could be… Continue Reading
Orchard Therapeutics Acquires GSK’s Gene Therapy Portfolio
Rare Daily Staff GSK will said it will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard… Continue Reading
NIH Researchers Establish Genetic Cause of Rare Bone Disorder
Rare Daily Staff Researchers at the National Institutes of Health uncovered a genetic basis of melorheostosis, a condition also known as… Continue Reading
FDA Approves Novartis’ Afinitor DISPERZ to Treat TSC-Associated Partial-Onset Seizures
Rare Daily Staff     The U.S. Food and Drug Administration approved Novartis’ Afinitor DISPERZ for the adjunctive treatment of adult… Continue Reading
Researchers Declare Breakthrough in Rare Brain Disease in Kids
Rare Daily Staff Researchers from Université de Montréal and the CHU Sainte-Justine and Université de Montréal Hospital say they have… Continue Reading