C3 is Increasing Scientific Collaboration to Fight LGMD2A/R1

Coalition to Cure Calpain 3 (C3) initiates scientific collaboration to bring the best minds together to fight limb-girdle muscular dystrophy type 2A (LGMD2A/R1). Working to cure LGMD2A/R1 requires connecting ideas, scientists, clinicians, patient groups and companies to elevate research to a new level. C3’s work actively promotes and cross-pollinates new science through collaboration with the goal of accelerating the development of treatments and even a cure in the near term. In celebration of ten years of progress in our mission to fund research for a cure and build global awareness, this week brings number seven in our ten highlights: C3 increases scientific collaboration in LGMD2A/R1.

To get to a cure faster, we need more science for rare diseases like LGMD2A/R1. C3 has been the catalyst for teamwork in cutting-edge science. We bring the LGMD2A/R1 research world together. Since our founding in 2010, we have hosted four elite scientific conferences dedicated to LGMD2A/R1. World-renowned experts from around the globe – neuromuscular clinicians, scientists from academia and industry, regulators, and funding agencies – have convened at the four conferences:

• Clinical Characteristics and Pathogenic Mechanisms of Calpainopathy: Santa Monica, California, 2011

• Clinical Characteristics, Pathomechanisms, and Trial Design for Calpainopathy: Naarden, the Netherlands, 2013

• Coalition to Cure Calpain 3 Scientific Meeting: Arlington, Virginia, 2018 (see photo)

• Gene Therapy for LGMD2A/R1 Workshop: Arlington, Virginia, 2019

These increasingly frequent LGMD2A/R1-focused meetings explore the newest developments and provide a platform for sharing new data, insights, and future possibilities. We are driving more unique and effective research.
C3 also commits valuable research dollars through grants that drive collaboration between research centers. Almost 30% of our research grants were awarded to projects that unite research from multiple institutions. An early success is “Defining Clinical Endpoints in LGMD,” a consortium of eight clinical centers that are working together to establish clinical outcomes for future clinical trials to help identify what investigational drugs are effective. With your help, we hope to expand and improve these combined efforts to get closer to a cure.

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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