Dr. Giles Campion, Prosensa’s Chief Medical Officer (https://www.prosensa.eu/) presented an overview of the latest drisapersen results in this morning’s patient webinar. An analysis of the Phase III data suggests that administering the drug earlier in the disease and treating longer can delay the progression of DMD. For example, in the 96-week extension data from the Phase III study, patients on continual treatment could walk 49 meters farther in a 6-minute walking test than those who took placebo for 48 weeks before switching to the drug. Dr. Champion also reported that a statistically significant (p<0.001) decline in creatine kinase, a potential marker of muscle cell damage, was observed at week 48 for the drisapersen group compared with placebo.
The company is currently working to complete the analysis of drisapersen results across all studies, and is in discussions with patient groups, clinical experts and regulators to decide the best path forward. Further dosing of patients is currently on hold pending a more complete analysis of the current data.
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