Status | Ongoing |
Funder | LGMD2I |
CI | Professor Volker Straub |
Sites | Newcastle |
More information |
|
The Global FKRP Registry is for patients with conditions caused by a mutation in the FKRP* gene. This mutation causes Limb Girdle Muscular Dystrophy type 2I (LGMD2I) and the rare conditions:
- Congenital Muscular Dystrophy type 1C
- Muscle Eye Brain Disease
- Walker-Warburg Syndrome
The Registry aims to:
- Help recruitment into clinical trials by identifying patients more readily
- Speed up research
- Provide more knowledge about the natural history and commonness of FKRP-related disorders
- Keep patients informed
The Registry was set-up in 2011 as an online patient driven registry. It is currently supported by the LGMD2I Research Fund.
*Fukutin-Related Protein