Limb-girdle muscular dystrophies (LGMD) are a heterogeneous group of genetically determined muscle disorders with a primary or predominant involvement of the pelvic or shoulder girdle musculature. More than 20 genes with autosomal recessive (LGMD2A to LGMD2Q) and autosomal dominant inheritance (LGMD1A to LGMD1H) have been mapped/identified to date.
Clinical trial on LGMD ongoing at the Institute:
- GNT-015-FKRP: Prospective, longitudinal study of the natural history and functional status of patients with limb girdle muscular dystrophy 2I
| Acronym | GNT-015-FKRP |
| Study type | RBM-HPS |
| Principal investigator | Tania Stojkovitck |
| Sponsor | Généthon |
| Fundings | Généthon |
| Study status | Ongoing |
| Recruitment status | Ongoing |
| Population | Adult |
| + infos on clinicaltrials.gov |
