The number of participants at this stage is very small. All those who are asked to join a trial are protected by regulations governing their participation. They must be fully informed and have had ample opportunity to ask questions and to think about their decision. All participants are free to leave a trial at any time without giving a reason, or can refuse to join it in the first place without jeopardising any other treatment they should be receiving. They have to sign a lengthy consent form confirming that all this has been made clear to them.
Clinical trials have to be exceptionally carefully set up and controlled and run.
Only then will there be broad agreement amongst clinicians and scientists based on reliable information. He set up a project to inject myoblast transfers into the big toes of boys with Duchenne muscular dystrophy. The results, he claimed, were very encouraging. Other clinicians and scientists had grave reservations about the validity of the trials which they believed to be premature and poorly structured. The Muscular Dystrophy Campaign in the UK advised families against going to the USA to take part.