Subscribe to
this category


A total of 463 posts are filed under Innovation
FDA Accepts Amicus Fabry Drug for Priority Review
Rare Daily Staff The U.S. Food and Drug Administration has accepted the New Drug Application and granted priority review status for Amicus… Continue Reading
Abeona Says Early Data from Ongoing Study Gene Therapy for Sanfilippo Shows Cognitive Improvement
Rare Daily Staff Abeona Therapeutics said updated clinical data from the ongoing phase 1/2 trial for its experimental gene therapy for the… Continue Reading
FDA Grants Zogenix Breakthrough Therapy Designation for Experimental Dravet Syndrome Therapy
Rare Daily Staff The U.S. Food and Drug Administration awarded Zogenix Breakthrough Therapy Designation for its investigational product,… Continue Reading
FDA Grants MeiraGTx Rare Pediatric Disease Designation to Gene Therapy for Eye Disease
Rare Daily Staff The U. S. Food and Drug Administration granted rare pediatric disease designation to MeiraGTx’s gene therapy product… Continue Reading
Kevan Chandler is Going Far
Kevan Chandler long dreamed of travelling. The problem was that many of the places he wanted to go weren’t wheelchair accessible. The… Continue Reading
Abeona Receives FDA RMAT Designation for Gene-Corrected Cell Therapy to Treat Epidermolysis Bullosa
Rare Daily Staff The U. S. Food and Drug Administration granted Abeona Therapeutics Regenerative Medicine Advanced Therapy designation for… Continue Reading
Alnylam Expands Access Program to Include Free Genetic Testing for People at Risk for AHPs
Rare Daily Staff Alnylam Pharmaceuticals said it has expanded the Alnylam Act program to include no-charge, third-party genetic testing and… Continue Reading

Follow us on Twitter