C3 is Connecting Patients to Clinical Studies

Coalition to Cure Calpain 3 is a vital hub connecting patients with limb-girdle muscular dystrophy type 2A (LGMD2A/R1) to clinical studies. In celebration of ten years of progress in our mission to fund research for a cure and raise global awareness, we are sharing highlights of our progress. This week, #6 in our top ten highlights, shows how we play a key role in facilitating groundbreaking LGMD2A/R1 science by connecting patients to clinical research studies.

C3 plays a vital role as a conduit for LGMD2A/R1 patients to actively play a part in scientific progress. Participating in clinical trials and research studies can accelerate the development of therapies for LGMD2A/R1, increase our knowledge about typical disease progression, and possibly lead to a cure.  

Through various efforts, C3 helps people like you or your loved ones be a part of the solution. We are positioned as the preeminent resource for patients and researchers – academic or industry – to find the right people to advance progress in LGMD2A/R1. We can help in two kinds of studies. In observational studies researchers follow the progress of your disease symptoms over time to better understand the course of disease in the absence of treatment. This can be used to set meaningful clinical endpoints and outcomes in future interventional clinical trials. Interventional trials test experimental drugs or treatments in people, sometimes for the very first time. This is the path to a cure.

To help trials find patients and patients find trials, C3 has developed the LGMD2A/R1 CLINICAL RESEARCH STUDY OPPORTUNITY RESOURCE. This includes educational material about clinical studies and a list of current opportunities for LGMD2A/R1 patients. The site is updated as new opportunities arise or the status of current trials change. There are studies underway to explore the progression, or natural history, of different forms of muscular dystrophy – including LGMD2A/R1. And when clinical trials begin for LGMD2A/R1 we will be the first to know.

Connecting patients to trials is also why our LGMD2A/R1 GLOBAL PATIENT REGISTRY is so important. Once registered, you will receive e-mail updates on research progress and clinical trial opportunities.

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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