Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

1 article from the last 30 days matching "cell therapy"

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 →

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases