Molecular treatments in genetic disorders is approaching. There are currently over 300 studies at various stages of translational efforts. Neuromuscular genetic disorders are on the top of the list. These studies are mostly being carried out in children. We aim to give a timely update in relation to clinical trials, followed by real life experience.

The principles yielded from clinical trials in pediatric neuromuscular disorders will have impact in other fields and topics such as hematology as already there is an approved gene therapy for hemophilia B. The discussion on molecular therapies to include gene transfer should now be regarded as ‘general culture’ in medicine. Physicians and other health care providers have been becomingly interested.

1. To heighten how clinical trials in rare genetic disorders are designed and conducted.
2. To understand emerging issues in real life experience
3. To reflect whether the current status will match expectations
4. To discuss possibilities and foresee the future