Professor Francesco Muntoni, professor of paediatric neurology at the Institute of Child Health and principal investigator said:
“We are very clear the clinical evidence presented in our press release is based on the scientific results of this study, and that these are the findings of an early trial phase.
“Our press release leads with the key finding of the production of the protein dystrophin in boys on the higher dose cohorts. Myself and colleagues in Newcastle were available to provide expert clinical overview on the research, and indeed provided a balanced and realistic summary of the outcome of the study to the press.
“We do not present our case studies as clinical experts, nor claim their subjective observations are based on scientific findings. They speak in their own words and provide a human dimension.
“In no way have we, nor would we want to, exaggerate the findings of this, or any study. In any case we don’t need to, we believe our findings to be an important step forward in finding a treatment for boys with this debilitating and life-limiting condition.
“This is good news.”
A point for 'further clarity if needed' was added to the original press release.
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Email: Coxs@gosh.nhs.uk
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