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.
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#rare-disease
All articles and blog posts tagged #rare-disease.
Articles
Blog
Blog
Reading Health Information About DMD Carefully
Health information about Duchenne can be misleading, exaggerated, or incomplete. Practical media literacy questions for families.
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.