Clinical Trials Review of Elevidys Gene Therapy: Is Elevidys Cost Worthy?
How effective is ELEVIDYS (delandistrogene moxeparvovec-rokl) gene therapy, which has been introduced to the pharmaceutical market, in relation to its cost of 3 million...
Our Call to Anvisa: All Brazilian Children Over 7 Should Receive Elevidys Gene Therapy
Duchenne Muscular Dystrophy (DMD) is a rare, life-limiting genetic disorder that primarily affects boys, leading to progressive muscle weakness and degeneration. The disorder has...
We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments
It’s heart-wrenching to think that children in today’s world, in countries across the globe, are losing their lives far too early simply because they...
Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments
While DMD families struggle to restore their children to health, pharmaceutical companies whose primary mission is to heal children are focused on year-end profits....
The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?
Why are DMD gene therapies so expensive? Over the last decade, significant advancements have been made in the development of gene therapies for DMD,...