Article
Storytelling, Awareness, and the Race Against Time in Duchenne Muscular Dystrophy
A conversation with advocate Thomas Bartlett on why storytelling and public awareness, as much as science, drive funding, policy, and access in Duchenne.
Tag
#policy
All articles and blog posts tagged #policy.
Articles
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.
Article
The Same Disease, Different Countries: What Duchenne Parents in America and Georgia Reveal About Access, Hope, and Time
A reported comparison of how Duchenne families in the United States and Georgia experience access, advocacy, and the urgency of time.
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
Insurance Appeals for Duchenne Families
Insurance appeals in DMD: when denials happen, how to structure the appeal letter, what evidence helps, and how patient organizations support families.
Blog
Financial Planning and Insurance for Duchenne Families
Practical guidance on insurance, disability benefits, copay assistance, equipment funding, and long-term financial planning for DMD families.
Blog
DMD Patient Advocacy History and Impact
Dmd patient advocacy history shows how families shaped research priorities, drug review, care standards, and access debates.
Blog
DMD Treatment Access by Country: Why It Varies
Dmd treatment access by country varies through regulators, payers, health technology assessment, pricing, and local budgets.
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
DMD Treatment Cost: Why Prices Are So High
Understand dmd treatment cost factors, including rare disease economics, manufacturing, evidence uncertainty, and payer decisions.
Blog
Accelerated Approval and Rare Disease Therapy
Accelerated approval pathways are central to modern rare disease therapy. They are useful, controversial, and often misunderstood.