C3 is Expanding the Field of LGMD2A/R1 Researchers

Coalition to Cure Calpain 3 (C3) is attracting more researchers to study – and ultimately cure – limb-girdle muscular dystrophy 2A (LGMD2A/R1). We have worked tirelessly to bring more scientists, research, and dollars to focus on and unravel our understudied disease. In celebration of ten years of progress in our mission to fund research for a cure and raise global awareness, this week brings number eight in our top ten highlights: C3 is growing the field of LGMD2A/R1 researchers.

Since our founding in 2010, a top priority has been to increase the number of scientists committed to researching and curing this orphan disease. LGMD2A/R1 has been neglected even by orphan disease standards. Our goal is to build a critical mass of scientists and ideas to help drive potential treatments and future approved drugs. In short, we need to create more shots on goal.

First, C3 continues to encourage young scientists in LGDM2A/R1 with our Early Career Investigator Travel Grant. This grant supports travel for scientists to attend and present important LGMD2A/R1 research at conferences. Our first Travel Grant helped to amplify groundbreaking research to use CRISPR gene-editing to repair mutations in CAPN3, the gene associated with LGMD2A/R1 disease. Dr. Stefanie Müthel, from the Charité Medical Faculty and the Max-Delbrück-Center for Molecular Medicine, presented this work at the Biology of Calpains in Health and Disease Conference in July 2019. Without this support, an important new scientific voice might not have been able to make the trip from Berlin to California for the conference.

Second, C3 continues to attract renowned researchers to the field of LGMD2A/R1 research. Research grants help attract the best and brightest to our field, such as Dr. Michele Calos, of Stanford University. Dr. Calos appied her expertise in DNA-mediated gene therapy to test its applicability in an animal model of LGMD2A/R1.

Third, C3 is an invaluable resource to aid and attract science to the field:

• We are committed to the creation and distribution of research tools
• We are an expert partner for researchers and companies considering entering the space
• We have our finger on the pulse of the LGMD2A/R1 field and are able to advise on many aspects including protocol design

To continue this important work, 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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