Henry J. Kaminski, MD, Professor of Medicine at The George Washington University describes the value of being in the Rare Diseases Clinical Research Network (RDCRN). Dr. Kaminsky leads the Dr. Kaminski leads the Myasthenia Gravis Rare Disease Network (MGNET), a consortium in the RDCRN.
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eyes, eyelids, facial expressions, chewing, talking, and swallowing. The condition is usually due to the presence of antibodies against acetylcholine receptors in the neuromuscular junction.
As noted by Dr. Kaminski, RDCRN has provided MG researchers with a framework to collaborate with other MG researchers so that they can learn from each other, not repeat themselves, and use the small population of MG patients at each site collectively to establish natural history and registry studies. These studies are also designed to improve the publication process. Dr. Kaminski noted that one recent study assessing the efficacy of performing a thymectomy in patients with early onset MG led to 10 papers being published and has spawned additional studies that in turn will elicit many more publications. Similar studies by other groups in the MGNET on targeted therapies, biomarkers, real-world outcomes, etc, are also underway that should lead to a more efficient means to understand this rare disease but improve how it is managed and treated.
Dr. Kaminski leads the Myasthenia Gravis Rare Disease Network (MGNET), a consortium in the Rare Diseases Clinical Research Network (RDCRN).
To learn more about MGNET visit reporter.nih.gov/project-details/9804343
To learn more about RDCRN, visit ncats.nih.gov/rdcrn
CheckRare is collaborating with RDCRN to educate health care professionals one ways they can be part of this network to advance clinical research. Visit our collaboration page at checkrare.com/rare-diseases-clinical-research-network/
