Building the First LGMD2A/R1 Patient Registry

Coalition to Cure Calpain 3 (C3) has launched the first and only limb-girdle muscular dystrophy type 2A (LGMD2A/R1) patient registry. In celebration of ten years of progress in our mission to fund research for a cure and raise global awareness, this week we are highlighting the importance of C3’s Global LGMD2A/R1 Patient Registry in all of our efforts.

Without an organized patient community, LGMD2A/R1 is likely to remain a neglected rare disease. A robust, up-to-date patient registry is a prerequisite to attract the attention of the scientific world to take up our cause in search of a cure. The C3 Global LGMD2A/R1 Patient Registry is one of the most important drivers for:

• Attracting the attention of researchers
• Highlighting the importance of finding a treatment or cure
• Populating clinical trials for promising therapies
• Showing researchers the many ways LGMD2A/R1 manifests and progresses

C3 is showing the way by building the Global LGMD2A/R1 Patient Registry, with valuable data and insights into the disease. The registry helps researchers, and all of us, better understand the number of people living with LGMD2A/R1 and the types, severity, and progression of symptoms of the disease. Our disease is a blank sheet of paper to many researchers, physicians, and pharmaceuticals companies. The registry helps bring the disease to life, putting human faces on LGMD2A/R1 with valuable insights for the how it progresses in different people and how it might be treated in the future.

The registry allows for the identification of a pool of potential patients eligible for clinical trials of promising studies. C3 anticipates our registry will be utilized in the future to recruit participants for interventional clinical trials. And it has already helped illuminate the disease better in natural history studies, to which we help patients connect.

Click here to register today!

If you are a patient, we highly encourage you to join our patient registry or keep contact information current.  And in the age of genomics we strongly recommend that participants upload their genetic reports – which can help researchers unlock the genetics associated with LGMD2A/R1. Our registry has a robust privacy policy. Your information will be stored in a secure electronic database. Personally identifiable data is never shared with a third party without obtaining permission from the registrant.

To continue this important work, we need your help. C3 has a pinpoint focus: to drive research for an LGMD cure. Every dollar counts to help grow support and research for this overlooked sub-type of muscular dystrophy.

Please donate now so that we can continue our strong momentum.  Through your generous financial support we can do even more in the next ten years to change the lives of patients in need.

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