Blog
Joining a Clinical Trial: What It Means for a Duchenne Family
Deciding whether a child joins a Duchenne clinical trial is hard. What participation involves, the hopes and burdens, questions to ask, and how families weigh it.
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#clinical-trials
All articles and blog posts tagged #clinical-trials.
Blog
Blog
Muscle MRI in Duchenne Muscular Dystrophy
Muscle MRI in DMD: fat fraction, T2 mapping, Mercuri grading. How imaging biomarkers track disease beyond cardiac and what they add to functional tests.
Blog
Patient Registries and Natural History Studies in DMD
DMD patient registries: CINRG, Duchenne Registry, TREAT-NMD, MD STARnet. What they do, who can join, and why enrollment supports the whole community.
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The DMD Research Pipeline: What's Beyond Approved Therapies
DMD research pipeline: CRISPR gene editing, next-generation gene therapy, exon skipping advances, anti-fibrotic approaches, and what families should know.
Blog
Functional Assessments in Duchenne Muscular Dystrophy
Functional assessments in DMD: 6MWT, NSAA, time-to-stand, PUL. What each measures, why they matter for trials and routine care.
Blog
DMD Clinical Trial: A Family Reading Guide
Use this dmd clinical trial guide to understand phases, endpoints, eligibility, placebo, risks, and questions for a care team.