Increase collaboration to widen access to DMD drugs, trials and care

When it comes to fighting a rare disease like Duchenne muscular dystrophy (DMD), it’s a matter of strength in numbers. Three Duchenne experts talk to us about how international collaboration between patients, pharma, clinicians and regulators could accelerate access to drugs, trials, and quality care. Dr. Paul Wexberg, of the Austrian Muscle Research Organisation, and John Gorman, of Duchenne Ireland, both have sons living with the condition. Réka Béndek is studying clinical psychology and is a volunteer at the Healing
Goodwill Foundation, Hungary’s patient advocacy group for people living with neuromuscular diseases. Change is afoot for people living with Duchenne, as a pipeline of potential new treatments drives innovation and awareness.