The Kurt+Peter Foundation, a nonprofit group that supports research into potential therapies, 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 affects children between the ages of four 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 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.”