French Observatory for Patients with Type 3 Glycogenosis

Launched by INSTITUT DE MYOLOGIE, FRANCE · Sep 26, 2024

Keywords

ClinConnect Summary

The French Observatory for Patients with Type 3 Glycogenosis is a clinical trial aimed at understanding Glycogen Storage Disease Type III (GSD-III), a condition that affects how the body stores and uses glycogen, a type of sugar. This disease can lead to problems like low blood sugar and muscle weakness, especially as people get older. The trial involves creating an online registry where information about patients with GSD-III will be collected. This will help researchers learn more about the disease's natural progression, how it affects patients, and what factors might influence the outcomes.

To participate in this study, patients must have a confirmed diagnosis of GSD-III based on specific genetic tests. The trial is open to people of all ages and genders who are willing to share their health information. By joining, participants will contribute valuable data that can lead to a better understanding of GSD-III, even though there is currently no specific treatment besides managing diet to help control symptoms. This study is an important step toward improving care and support for those living with this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Patients with molecularly characterised Glycogen Storage Disease Type III
• Exclusion Criteria:
• • Patients diagnosed with GSD type 3 refusing to take part in the study