Pfizer has announced that its mini-dystrophin gene therapy has not met its primary or secondary endpoints. Click here to read Pfizer’s update on Phase 3 of CIFFREO, a randomised double-blind placebo-controlled study in ambulatory boys aged 4-7. The primary...
Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...
