Coalition to Cure Calpain 3 in Partnership with the National Organization for Rare Disorders (NORD®) Launches LGMD2A/Calpainopathy Registry
WESTPORT, Conn., Sept. 21, 2023 /PRNewswire/ -- Today, Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare form of muscular dystrophy that causes progressive muscle weakness and wasting. Calpainopathy currently has no cure.
The new study, the LGMD2A/Calpainopathy Registry, creates a platform to bring the Calpainopathy community together and collect patient data that is an essential requirement for policy makers, academic researchers, and therapeutics companies to advance treatments for this disease. Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials.
The LGMD2A/Calpainopathy Registry replaces C3's original patient registry. While the original registry served primarily to contact Calpainopathy patients, the new registry will have the capability to collect longitudinal data by having participants periodically update their information. This information is vital to help us understand the progression of symptoms over time, which will inform patient care, drug development, and clinical trial design. "Our goal is to enroll as many patients, or their parents or legal guardians, as possible," notes Jordan Boslego, C3 President. "The success of the registry is dependent upon community participation, and it's essential that the 1000+ patients in our current Global LGMD2A Registry take the time to register on this new platform."
"The LGMD2A/Calpainopathy Registry will provide a complete picture of each patient's experience with this disease," shares Dr. Jennifer Levy, C3 Scientific Director. "We are launching this initiative to help fill in the missing information researchers and medical experts need to advance research and one day, find a cure."
"This new study has tremendous promise as a strong partnership that engages the patient community and addresses current knowledge gaps for Calpainopathy. NORD is thrilled to be a part of driving research and innovation-based outcomes for all the families in the Calpainopathy community," says Aliza Fink, Director of Research Programs, NORD.
For more information, visit https://LGMD2A.IAMRARE.ORG.
About Coalition to Cure Calpain 3
Coalition to Cure Calpain 3 (C3), a U.S.-based, public, 501(c)(3) non-profit organization, was founded in 2010 to fulfill a mission to drive high-quality research while educating the global community about the muscle-wasting disease limb-girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. C3 has committed over $2 million in grants to international leaders in the muscular dystrophy field to support the development of essential research tools, the identification of outcome assessments that will be used in future clinical trials, and the testing of potential therapies in animal models to determine if they can be safe and effective for LGMD2A. For more information on C3's efforts to treat and cure this disease, visit https://www.curecalpain3.org.
About National Organization for Rare Disorders, Inc. (NORD®)
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit http://www.rarediseases.org.
Media Contacts
Jennifer Levy, C3
[email protected]
(203)429-4383
Marketing, NORD
[email protected]
SOURCE Coalition to Cure Calpain 3
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