C3 Celebrates a Decade of Achievement

Coalition to Cure Calpain 3 (C3) has come a long way in ten years in driving science and awareness to treat limb girdle muscular dystrophy, type 2A (known as LGMD2A/R1, LGMD R1 Calpain 3 related, or calpainopathy). In celebration of ten years of progress in our mission to fund research for a cure and build global awareness, C3 will be highlighting ten areas of momentum over the next ten weeks. We will also be asking for your support to keep this progress going so that we might see a cure from our next decade of work.

C3 is a non-profit organization founded by Kristine Kurnit and Michele Wrubel, two women living with LGMD2A/R1. Prior to 2010, there was no organization dedicated specifically to understanding and curing this disease. LGMD2A/R1 is one of many different diseases encompassed by umbrella organizations, such as the Muscular Dystrophy Association. Our co-founders recognized that LGMD2A/R1 needs more research and attention. As a rarer and sometimes less-severe form of muscular dystrophy, it attracts significantly less research funding and scientific investigation. Nevertheless, it still has an enormous impact on patients’ lives.

That is why we are driving high-quality research with the goal to develop a cure faster. To drive the science forward, C3 continues to build the essential tools and understanding necessary to attract and support scientists and research. Ultimately our goal is for biopharmaceutical companies to see potential in developing a treatment for this disease.

• Awarded more than $1.4 million in grants to international leaders in the muscular dystrophy field

• Developed four new animal models to facilitate preclinical research; these are commercially available from Jackson Laboratory

• Supporting a study to identify clinical outcome assessments, which will be used in future clinical trials to determine whether an investigational therapy benefits LGMD2A/R1 patients

• Funded crucial basic research elucidating the biology of LGMD2A/R1 and how it affects the body

• Fostered preclinical testing to assess whether potential therapies, including gene therapy, can be safe and effective for LGMD2A/R1 patients

We need your help. C3 has a pinpoint focus: to drive research for an LGMD2A/R1 cure. Every dollar counts to help grow support and research for this overlooked subtype 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 10 years to change the lives of patients in need.

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