MENLO PARK, Calif., Dec. 17, 2013 /PRNewswire/ -- The Kurt+Peter Foundation (www.kurtpeterfoundation.org) announced today that it has released the first patient registry for Limb Girdle Muscular Dystrophy, Type 2C, also known as gamma sarcoglycanopathy (LGMD2C). Recent scientific developments suggest that new treatments for LGMD2C may be ready for testing in patients in the next few years. The patient registry will allow researchers to find patients for clinical studies.
LGMD2C is a severe disease that typically impacts children between the ages of 4 and 12. Children with the disease lose muscle strength and typically lose the ability to walk by their teens. Medical advances, such as exon skipping, high-throughput screening of existing drugs, and improved prospects for gene therapy, offer hope for treating LGMD2C.
"Recent research provides reason for optimism that clinical trials for therapies for LGMD2C are not too far in the future," said Dr. Elizabeth McNally of the University of Chicago, a leading LGMD2C researcher. "A registry such as the one created by the Kurt+Peter Foundation will be important for identifying and registering patients."
The LGMD2C registry is available at both www.lgmd2cregistry.org and www.kurtpeterfoundation.org/patient_registry . It will allow patients to register to provide information that researchers will need for potential clinical trials.
"We are excited to have developed and released this registry that will allow patients to register for research for an underdiagnosed and severe disease that impacts children worldwide," said Scott Frewing, president of the Kurt+Peter Foundation. "The registry will allow clinicians to identify patients and share that information to assist clinical trials," he continued.
About the Kurt+Peter Foundation
The Kurt+Peter Foundation is a nonprofit group that supports research into potential therapies for LGMD2C. The foundation's mission is to expedite treatments to help this generation of children. The charity was founded in 2010 by Dr. Charlotte Drew and Scott Frewing, whose 6- and 8-year-old sons have LGMD2C. To learn more visit www.kurtpeterfoundation.org.
LGMD2C is a severe form of muscular dystrophy with symptoms similar to Duchenne muscular dystrophy. Children with LGMD2C cannot produce a protein necessary for muscle strength and function. Muscles deteriorate instead of growing stronger. Although children with LGMD2C are able to live normally when young, their muscles deteriorate quickly, limiting childhood activities.
Press contact: Scott Frewing
SOURCE Kurt+Peter Foundation