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Risks and benefits

Risks and benefits of participating in a clinical trial

When you are looking for treatment of Duchenne or Becker muscular dystrophy, you might want to join a clinical trial. In this type of research study, you play an active role in a decision that affects the live of your child. Although clinical trial participation comes with possible benefits, it also carries possible risks that need to be considered before joining.


of participating in a clinical trial

  • It might not be better, or even as good as, current treatment standards
  • There may be costs, such as travelling or accommodation, involved you have to pay yourself
  • Although potentially beneficial to others, it might not work the same for you
  • You will need to spend more visits to a hospital/clinic taking tests and being monitored
  • You will have to see your clinician more often
  • There is a possibility that you and your family have to live abroad if a trial site is in another country
  • During treatment, side effects can occur that were not expected
  • To test efficacy, sometimes uncomfortable tests need to be taken.
  • You may be excluded to participate in other trials
  • In a randomized clinical trial, there is a possibility that you will receive a placebo instead of the drug, possibly not benefiting the potential effectivity

There are many risks and benefits that need to be addressed before starting a clinical trial. Your site manager can answer your questions.


of participating in a clinical trial

  • If the drug is effective, your child will be the first to benefit from it
  • You have access to new types drugs that are not available yet for people outside the trial
  • Your health will be closely monitored by a professional research team
  • You will contribute to research that will help scientists learn more about Duchenne or Becker muscular dystrophy

Its important to remember that there is a chance you or your child might not benefit from the clinical study directly. There are certain study types designed to gain general knowledge about the condition. The argument ‘They asked me to participate because this trial is made for people like me or my child’ is called the therapeutical misconception.

Make sure that you understand all the ins and outs of the study design. Discuss your choice with an independent clinician or your own general practitioner. You are always free to refuse or withdraw from a study without consequences for the care you are receiving.