Objective: align on strengths and weaknesses and outstanding questions identified by the groups
Objective: introduce how and why clinical trials are conducted, the objective of different trial phases, trial design, primary endpoints, secondary endpoints, clinical significance, ethical concerns and informed consent
Participants will be provided with a scenario for a clinical trial plan for a drug to be tested in children. Different roles will be given to different participants.
Objective: gain insight in ethical discussions related to clinical trials and the perspectives of different stakeholders
Objective: overview of the different types of functional outcome measures used in clinical trials for neuromuscular, their opportunities and limitations, need for standardization and training, TREAT-NMD efforts in their area, natural history studies and their usefulness in trial design
Objective: to illustrate the consequences of using outcome measures limited to certain disease stages with regards to extrapolation and limited indications
Objective: to outline the steps and stakeholders involved in developing and validating functional outcome measures that assess what patient find important using upper limb function outcome measure development for DMD and SMA as showcases
Objective: to gain insight in available tools and services for planning and conducting clinical trials