Clinical Studies
For SARCOGLICANOPATHies
2024
Natural history study by Atamyo Therapeutics for LGMD2C-R5. It takes place in France and Tunisia.
To take part in the project, participants must visit the clinical centers every 6 months.
More information
2021
Since 2021, the observational study Quality by the GFB Foundation on the quality of life of patients with Sarcoglycanopathy (alpha-sarcoglycan, beta-sarcoglycan, and gamma-sarcoglycan mutations) has been active.
The project recruits patients on all continents and is carried out directly from home, using various communication methods, including email, social media, and postal mail.
2020
Since 2020, the natural history study Journey by Sarepta Therapeutics for Sarcoglycanopathies (LGMD2C or LGMDR5 - LGMD2D or LGMDR3 - LGMD2E or LGMDR4) has been underway. It takes place in Europe, the United States, Canada, Brazil, Turkey, and the United Kingdom.
To take part in the project, participants must visit the clinical centers every 6 months.
2018
Since 2018, two natural history studies for LGMD2E or LGMDR4 have been ongoing, one in the United States and one in Milan, Italy.
United States
It is conducted by Prof. Jerry Mendell at Nationwide Children's Hospital in Columbus, Ohio. To participate, patients must periodically visit the clinical center.
Milan
It is conducted by the GFB Foundation and Prof. Yvan Torrente, University of Milan and Policlinico. The project recruits only Italian patients with Sarcoglycanopathy and is carried out directly from home, using various communication methods, including email, social media, and postal mail.