28/09/2026
WHAT DOES “CLINICAL TRIAL” ACTUALLY MEAN?
Researchers study the potential treatment in laboratory and other preclinical models before it can move into human testing.
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1️⃣ PHASE 1
“IS IT SAFE?”
Researchers primarily look at safety, side effects and appropriate dosing when the treatment is first tested in humans.
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2️⃣ PHASE 2
“DOES IT SHOW SIGNS OF BENEFIT?”
Researchers continue monitoring safety while gathering preliminary evidence about whether the treatment may help people with the condition.
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3️⃣ PHASE 3
“DOES THE EVIDENCE HOLD UP?”
Larger studies gather more information about effectiveness and safety and help establish the overall benefit-risk profile.
🟢 IMPORTANT TO REMEMBER
A clinical trial is not the same thing as an approved treatment.
A treatment being tested in a trial is still being investigated. The purpose of clinical research is to collect evidence and determine whether an approach is sufficiently safe and effective for its intended use.
And in DMD, researchers are currently investigating a range of approaches, including strategies involving dystrophin restoration, exon skipping, muscle protection and other molecular therapies.
