Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.
Giulia RicciLuca BelloFrancesca TorriErika SchirinziElena PegoraroGabriele SicilianoPublished in: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology (2022)
In the last 20 years, research efforts converged in characterization of the disease mechanism and development of therapeutic strategies. Same effort needs to be dedicated to the development of outcome measures able to capture clinical benefit in clinical trials.