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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.
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#regulation
All articles and blog posts tagged #regulation.
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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.
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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.
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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.
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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.
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Health Technology Assessment in Rare Disease
Health technology assessment shapes which rare disease therapies are funded, and how. A primer for families and advocates.
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Accelerated Approval and Rare Disease Therapy
Accelerated approval pathways are central to modern rare disease therapy. They are useful, controversial, and often misunderstood.