Article
The Duchenne Lottery: Why Where a Child Lives May Now Matter More Than Ever
ELEVIDYS, the first Duchenne gene therapy, is approved in the US and Japan but rejected in Europe, leaving a child's access increasingly shaped by geography.
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#regulation
All articles and blog posts tagged #regulation.
Articles
Article
Duchenne, Drug Approval, and Public Policy: Why Georgian Families Are Demanding Action
How regulators, payers, and politics meet in the Duchenne treatment access dispute in Georgia, and what it says about rare disease policy.
Blog
Blog
Vamorolone (Agamree): A Modified Steroid for DMD
Vamorolone is a glucocorticoid receptor modifier approved for Duchenne. It aims to preserve anti-inflammatory benefit while reducing some side effects.
Blog
Givinostat (Duvyzat): First Non-Steroidal DMD Treatment
Givinostat, marketed as Duvyzat, is the first non-steroidal DMD drug approved by the FDA. What the science, the trial, and the label actually say.
Blog
Elevidys Explained: The First DMD Gene Therapy
Elevidys is the first FDA-approved gene therapy for Duchenne muscular dystrophy. What it is, who it is for, and what the regulatory record actually says.
Blog
How to Read Regulatory News About DMD
Regulatory news about DMD therapies can be confusing. A practical guide to reading FDA, EMA, and post-marketing safety updates carefully.
Blog
Health Technology Assessment in Rare Disease
Health technology assessment shapes which rare disease therapies are funded, and how. A primer for families and advocates.
Blog
Accelerated Approval and Rare Disease Therapy
Accelerated approval pathways are central to modern rare disease therapy. They are useful, controversial, and often misunderstood.