Mesoblast (ASX:MSB) has received clearance from the US Food and Drug Administration to proceed directly to a registrational trial of its therapy, Ryoncil, in children with Duchenne muscular dystrophy.
Duchenne muscular dystrophy, or DMD, is caused by the absence of dystrophin, a protein essential for maintaining muscle integrity. The disorder leads to gradual muscle degeneration, ultimately affecting mobility, breathing, and heart function. Most patients, primarily boys, face a dramatically shortened lifespan, often not surviving beyond early adulthood.
In DMD, inflammation accelerates muscle damage, compounding the effects of dystrophin deficiency.
Ryoncil, a mesenchymal stromal cell therapy already approved for a separate paediatric condition, is designed to modulate this inflammatory response.
Seventy-six boys between the ages of five and nine will participate in the trial, receiving either the therapy or a placebo alongside standard care. Over nine months, researchers will track their ability to rise from a seated position, a functional milestone that serves as a meaningful indicator of disease progression and has been accepted by regulators as a valid endpoint.