According to the Rare Clinical Diseases Research Network (RDCRN) website, the purpose of the RDCRN is to increase collaboration between the research and treatment efforts of the scientists and clinicians working with many different types of rare disease. The RDCRN receives funding from the National Institutes of Health (NIH) and Office for Rare Diseases Research in the U.S.A.
The RDCRN also works to improve the availability of information and treatment to patients. By registering with the RDCRN contact registry, individuals will receive disease-specific information via email regarding open recruitment for clinical research, new clinical research sites, and activities related to raising awareness of specific neuromuscular diseases and advocacy efforts. The website also contains information regarding each of the included diseases.
The RCDRN originally included five research consortia. In 2009, the Network received funding to expand the network to include an additional 14 consortia covering 95 rare diseases. To be considered a ‘rare disease’ by the RCDRN, the disease must affect less than 200,000 individuals in the U.S.A.
A number of the neuromuscular diseases meet criteria for inclusion as a rare disease and are represented by the RCDRN. These include the mitochondrial diseases, inherited neuropathies including Charcot Marie Tooth disease, episodic ataxias, non-dystrophic myotonic disorders, and lysosomal storage disease including Pompe disease. Three of the groups added in 2009 are headed by researchers that have been supported by the Muscular Dystrophy Association (MDA).
According to the MDA, before registering with any of the disease registries, individuals should take a number of things into consideration. These include: the purpose of the registry; how much time registration will take; how the registry is funding and who oversees the registry; how the registry will benefit registered individuals; and how privacy is protected. Red flags that may lead one not to register may include lack of privacy protection, requests for information about finances, lack of clear purpose, and difficulty getting answers to questions.
Registering with the RDCRN was simple, and took me less than minutes. After reading and agreeing to the terms in the authorization form, I was asked for personal information, such as name, address, and birth date, and regarding my diagnosis. According to the website, data storage is secure, and privacy protected. By registering with the RDCRN, individuals can help to further the work of researchers looking for more effective treatments and cures for neuromuscular disease.
CMTA, (2011). The RDCRN – A Valuable Resource for CMT Patients. CMTA website. http://cmtausa.org/index.php?option=com_content&view=article&id=330:the-rdcrn-a-valuable-resource-for-cmt-patients&catid=7:newsflash&Itemid=61 . Retrieved 2/23/12.
Muscular Dystrophy Association, (2009). Muscular Dystrophy Association Applauds New Federal Support For Neuromuscular Diseases. MDA website. http://www.mda.org/news/091008Federal%20Support.html . Retrieved 2/23/12.
Rare Clinical Diseases Research Network, (n.d.). Rare Clinical Diseases Research Network website. http://rarediseasesnetwork.epi.usf.edu/index.htm . Retrieved 2/23/12.
Wahl, M., (2006). To Register or Not to Register. Quest, 13:6. http://quest.mda.org/article/register-or-not-register . Retrieved 2/23/12.