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The information contained in the Organizational Database (ODB) is provided for informational purposes only. There is no implied endorsement by NORD. NORD does not promote or endorse participation in any specific organization. The information is subject to change without notice. Every effort is made to ensure that the details for each entry are as current as possible.

Global FKRP Registry

Address

TREAT-NMD Office
Institute of Genetic Medicine
Newcastle upon Tyne, NE1 3BZ
United Kingdom

Phone

440-191-2418617

Fax

440-191-2418770

Email Address

coordinator@fkrp-registry.org

Website

https://www.fkrp-registry.org/

The Global FKRP Registry is the first international collection of data for both patients and professionals involved in the care of individuals with conditions caused by mutations in the fukutin related protein (FKRP) gene. This includes people with the type 2I form of limb girdle muscular dystrophy (LGMD2I) and the type 1C form of congenital muscular dystrophy (MDC1C) and, in rare instances, the congenital muscular dystrophies muscle-eye-brain disease and Walker-Warburg syndrome. FKRP-related conditions are rare, thus without a patient registry to gather details in one place from all FKRP patients worldwide, finding enough patients for a meaningful trial can take years. The web-based Registry aims to facilitate the identification of potentially eligible patients for future clinical trials of new therapies, accelerate research into new therapies for these conditions, keep patients informed about research results, and provide more detailed knowledge about the prevalence and natural history of FKRP-related conditions.

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