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2 articles from the last 7 days matching "dmd"

Clinical trialRSSYesterday

How 19 Points Changed My Grandson’s DMD Journey

A grandmother shares how a small change in how her grandson's condition was measured helped him qualify for a Duchenne muscular dystrophy (DMD) treatment trial. Researchers adjusted the scoring system by 19 points, which gave him a second chance to participate in the study. This story highlights how trial eligibility rules can sometimes be adjusted to help more patients access new treatments.

WHY IT MATTERSIf your child with DMD has been told they don't qualify for a trial, this story shows that eligibility criteria can sometimes be reconsidered or adjusted—it's worth asking your doctor whether similar flexibility might apply to your situation.
💬 Ask your doctorDuchenne muscular dystrophyRead →
Clinical trialRSS3 days ago

FDA clears trial testing Mesoblast cell therapy in kids with DMD

The FDA has approved a clinical trial for a new cell therapy called remestemcel-L-rknd made by Mesoblast. The therapy will be tested in children ages 5-9 who have Duchenne muscular dystrophy (DMD), a serious muscle disease. About 76 children will participate in the trial and receive either the therapy or a placebo while continuing their regular DMD treatments.

WHY IT MATTERSThis trial represents a new treatment approach for DMD in young children who are still in a critical window for muscle development, offering families a potential alternative or complement to existing therapies.
You can act on thisDuchenne muscular dystrophyRead →

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