Disease registries have been formed to aid researchers searching for qualified individuals to participate as subjects in research protocols. Such research is needed to advance knowledge regarding the diagnosis and treatment of neuromuscular disease. In turn, enrolling in a registry specific to neuromuscular disease will help individuals to be informed about clinical research regarding a specific disease or condition, and some of the registries also provide disease-specific information to enrolled individuals.
The North American Mitochondrial Disease Consortium, established by the U.S. National Institute of Health and part of the Rare Clinical Diseases Research Network, collects information about individuals who have been diagnosed with mitochondrial disease and their family members. Those enrolled in this database must enroll in the RCDRN (you can find the website address above) and be willing to travel to one of the participating centers for an annual visit, a list of which can be found at https://rarediseasesnetwork.epi.usf.edu/NAMDC/centers/index.htm .
Recently, a couple of new research registries related to neuromuscular disease have been announced. Individuals with Becker Muscular Dystrophy (BMD) may now register with research registry related to the study of the follistatin gene transfer study at Nationwide Childrenís Hospital in Columbus Ohio. Researchers have been conducting clinical research regarding the safety of a treatment (involving injection of a gene transfer protein follistatin into the thigh) for individuals with BMD and sporadic inclusion-body myositis. For more information about the registry and this and other research, see the hospitalís website: http://www.nationwidechildrens.org/gene-therapy-clinical-studies .
A registry for individuals with ataxia, including Friedreichís ataxia, has been opened through the Coordination of Rare Diseases at Sanford (CoRDS). Eligible individuals can find out more at http://www.sanfordresearch.org/cords/faqs/participantfaq/ .
For more information and additional registries, see the article referenced below, Research Registries and Neuromuscular Disease.
Madsen, A., (2013). Ataxia Registry Open for Enrollment. Quest. Retrieved from http://quest.mda.org/news/ataxia-registry-open-enrollment on 8/8/13.
Reijonen, J., (2012). Research Registries and Neuromuscular Disease. Retrieved from http://www.bellaonline.com/articles/art178947.asp on 8/8/13.
Reijonen, J., (2012). The Rare Clinical Diseases Research Network. Retrieved from http://www.bellaonline.com/articles/art175332.asp on 8/8/13.
Wahl, M., (2013). BMD: Participants Sought for Registry Associated with Gene Transfer Trial. Quest. Retrieved from http://mail.aol.com/37966-111/aol-6/en-us/mail/DisplayMessage.aspx?ws_popup=true on 8/8/13.
Wahl, M., (2012). Mitochondrial Disease Registry Seeks Participants. Quest. Retrieved from http://quest.mda.org/news/mitochondrial-disease-registry-seeks-participants on 8/8/13.