Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) has launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare form of muscular dystrophy that causes progressive muscle weakness and wasting. Calpainopathy currently has no cure.
The LGMD2A/Calpainopathy Registry is a global platform designed to unite the Calpainopathy community and gather crucial patient data. This registry will be a critical resource for policy makers, academic researchers, and therapeutic companies working diligently to advance treatments for Calpainopathy. It will also be collecting data that is aligned with the TREAT-NMD LGMD Core Dataset. This means that the data collected will adhere to high-quality standards, making it invaluable for research and treatment development. TREAT-NMD is also pleased to support this project by the roll out of the registry being partially funded by the TREAT-NMD LGMD bursary scheme.
The registry will also serve as a conduit to identify individuals with Calpainopathy who may be willing to participate in other research studies or clinical trials. By fostering collaboration and increasing research participation, the aim is to accelerate the pace of discovery and innovation in the field of Calpainopathy.
Replacing C3’s original patient registry, the LGMD2A/Calpainopathy Registry offers more than just a means of contact. It empowers participants to periodically update their information, enabling the collection of longitudinal data. This longitudinal data is vital as it provides invaluable insights into the progression of symptoms over time. Such knowledge is instrumental in shaping patient care, driving drug development, and designing clinical trials.
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