Goals, Mission & Vision h1 >
Our Mission h1 >
The challenge for the NAMDC is the extraordinary clinical spectrum of mitochondrial diseases, which all too often leads practitioners to either underdiagnose ("What is this complex disorder?") or over diagnose ("This disorder is so complex that is must be mitochondrial!"). Yet mitochondrial diseases cause similar metabolic defects and presumably share—albeit to different extents—the same mechanisms. Thus, the availability of a mitochondrial patient registry and of a consortium will have a powerful impact in multiple ways, as already documented by similar organizations operating in Europe.
First, the NAMDC will make these rare and still unfamiliar diseases known to practitioners and to the general public.
Second, it will facilitate correct diagnosis by making "centers of excellence" available to physicians and affected families alike.
Third, it will offer affected families the comfort and advice of a patient support group, the United Mitochondrial Disease Foundation (UMDF).
Fourth, it will foster clinical research, such as natural history, that would be otherwise impossible because it requires relatively large cohorts of patients.
Fifth, it will also foster more basic research by revealing unusual patients, leading to the discovery of new genetic defects.
Finally, the NAMDC will conduct rigorous and innovative therapeutic clinical trials.