What is Known:

• When planning and conducting clinical trials, sponsors and researchers know that clinical trials are to be performed according to well-established ethical rules, and patients should be aware about their rights.

• In the cases of paediatric patients, vulnerable patients unable to provide consent, genetic diseases’ further rules apply.

What is New:

• This work discusses which ethical rules apply to ensure protection of patient’s rights if all the above-mentioned features coexist.

• This work shows available data and information on how these rules have been applied.