Rare disease registries provide an abundance of critical data and resources
An article in Contemporary Clinical Trials asks the pertinent question: If you build a rare disease registry, will they enroll and will they use it? The authors, from the National Registry of Myotonic Dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD) answer the question affirmatively and go on to underscore the overwhelming value of rare disease registries to gather patient populations for clinical studies and for many other uses: to develop collaborative investigations, learn more about the natural history and pathophysiology of the disease(s), gather and compare genetic data; assess the impact of existing treatments; study the economic impact of the disease and treatment; and capture data on quality of life and psychosocial issues. The authors discuss their strategies for enrolling patients.
