International Registry Expands to Include Congenital Muscle Diseases
The congenital muscle disease international registry (CMDIR) will play a vital role in linking individuals with researchers and clinicians. Two years ago, Cure CMD, an organization dedicated to finding treatments for children with congenital muscular dystrophy, launched the first and only congenital muscle disease international registry (CMDIR). The registry’s prime objective was to identify people with this disease, improve standards of care, and most importantly, prepare for clinical trials to find potential drug treatments.
To reach even more people with similar conditions, the international registry has expanded to collect key information from people affected by congenital myopathies, thanks to the successful partnership of several congenital muscle disease advocacy groups
“We’re very excited about this expansion,” explained Anne Rutkowski, chair and co-founder of Cure CMD. “Finding more people with these diseases will enable us to create a critical mass to participate in clinical trials. Simply put, we will not be successful in finding a treatment or cure for these diseases unless we know who the affected individuals are, what the diagnosis is and how the disease is affecting the individual.”
“Patient registries provide an essential and important interface between patients, clinicians and researchers”, added Dr. Kathryn North, professor at the University of Sydney in Australia. “Expansion of the registry for congenital muscle disorders is essential in raising standards of care for this rare group of conditions and ensures equity of access to clinical trials for all patients.”
The registry includes demographic, health and diagnostic questions. Its mission is to act as a central hub for up to date clinical study and trial information, collect data on baseline medical information and treatment interventions and support genetic confirmation through referral to national centers of diagnostic excellence. The registry is available in English, Spanish, French, Portuguese and German.
“In Australia, State Muscular Dystrophy Associations, are actively spreading the news about the CMDIR to their Clients”, said Rick Carr, CEO of Muscular Dystrophy Queensland. “By participating in this international registry, our Clients can assist worldwide research seeking to unravel the mysteries of these degenerative conditions”.
To register, simply log on to WWW.cmdir.org and click the ‘register now’ button.