French study on type 3 Glycogen storage disease

This study looks at the natural history (how the illness typically progresses) in people with GSD VI or GSD IX. It may help doctors better understand disease patterns, which can support future treatments.

This study follows people with rare types of glycogen storage disease (GSD) over time to learn more about how the disease progresses. It does not test a new treatment but will collect information to help future research.

This study is creating a registry (a database) to better understand glycogen storage diseases in Indian children. If your child has a confirmed genetic diagnosis, joining helps doctors learn more about the condition and improve care.

This trial tests whether a medicine called diazoxide can help treat type 1 glycogenosis, a rare metabolic condition that affects how the body stores and uses sugar. Participation is limited to patients already being followed at one of two French hospitals.

This study looks at people with Gaucher disease (types I, II, or III) to better “group” patients for understanding and future research. If you (or your child) have confirmed Gaucher disease, you may be asked to share information and follow a study schedule.

This study tests a new gene therapy, VGN-R08b, for people with type 3 Gaucher disease. It aims to see if the treatment is safe and can help with neurological symptoms like eye movement problems.