Cure CMD, the LGMD2I Fund and the Newcastle TREAT-NMD office announce a collaboration to promote clinical trial readiness for LGMD2I. In anticipation of an LGMD2I clinical trial, we are working to align the 2 existing registries, one specific for FKRP and one for each of the other CMD-LGMD alpha dystroglycan related dystrophy (aDG-RD, dystroglycanopathy) genes. To launch a specific LGMD2I trial, we will need to have all FKRP patient information in one location, the FKRP international registry.
We would like to ask all people with confirmed FKRP mutations to register in the international FKRP registry (www.fkrp-registry.org). We would like to ask all people with a confirmed mutations in each of the other aDG related genes, including POMT1, POMT2, POMGnT1, DAG, LARGE and fukutin and those without a genetic diagnosis to register in the CMD International Registry (CMDIR-www.cmdir.org).
Both registries will continue to work together to provide registrants with the same up to date information on clinical studies and trials in the aDG-RDs. We believe that strides made in clinical trial readiness for the LGMD2I population will have significant impact for the larger group of patients with alpha-dystroglycan related dystrophies (aDG-RDs). We would like to thank all who have currently registered and encourage registrants to fully complete the survey questions.