Myasthenia Gravis Rare Disease Network |  |
Enhancing discovery and treatment for myasthenia gravis (MG), a neuromuscular disorder caused by an autoimmune response which blocks or damages acetylcholine receptors in muscles, causing disabling weakness. | |
Principal Investigator
George Washington University
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TOP PUBLICATION
JOURNAL: Annals of Neurology
DOI: 10.1002/ana.78017
Patients with early-onset MG develop symptoms before age 50, while patients with late-onset MG develop symptoms after age 50. Not much is known about how early-onset and late-onset MG affect the body differently.
Utilizing sera (the liquid part of blood that remains after coagulation) from the NIH-sponsored BeatMG clinical trial and MGNet, researchers performed proteomics analysis of 768 inflammatory proteins and uncovered a distinct immunological signature that differentiates late-onset from early-onset myasthenia gravis.
Pathways linked to leukocyte differentiation and myeloid cell migration were enriched in late-onset disease. Seven proteins were further replicated in the UK Biobank, and IL18R1, CXCL17, and CCL11 were validated in an independent cohort that were specific to late-onset MG. Investigators note that further studies are needed to confirm the use of these proteins as biomarkers of late-onset MG, as well as their use for improving therapeutic precision and patient outcomes.
What was your consortium's most significant achievement from the past year?
Henry Kaminski, MD, Principal Investigator, MGNet: We have established the MGToolKit in Epic to collect research-grade data in the context of routine clinic visits. We have also recruited the first subjects.