It has been estimated that more than one third of patients with neurological disorders with problems affecting the neuromuscular system. Striking progress has been made in understanding many of these diseases, and in the effective application of the new knowledge to treatment. This progress has been made possible by the intimate interaction of basic and clinical scientific approaches. At the same time, the analysis of human neuromuscular diseases has contributed to the understanding of the basic biology of nerves, muscles, and nerve-muscle interactions. The goal of this proposal is to foster the training of both clinical and basic scientists in the areas of neuromuscular biology and disease, so as to encourage even more intense interactions between these approaches. The faculty, laboratories and programs involved are well-established, highly interactive, and productive. The areas of research focus on the neuromuscular system, but entail a wide range of approaches and methodologies. Representative disease-oriented areas include: amyotrophic lateral sclerosis; spinal muscular atrophy; peripheral neuropathies; myasthenia gravis; muscular dystrophies; and autoimmune myopathies. A wide variety of research approaches are used, and basic methods include those of molecular biology, immunology, morphology, pharmacology, cell culture, and electrophysiology. Clinical material and clinical trials serve as lynch pins of the program. The trainees will be both clinicians and basic scientists with doctoral degrees, (many MD/PhDs) whose goals are to perform significant research in the fields of neuromuscular diseases and biology; Support for four postdoctoral fellows is requested is requested. We will stress the recruitment of minority applicants. The training program involves intensive laboratory research, and postdoctoral fellows are encourage to audit relevant courses and to participate in the well-organized programs on seminars and conferences. Special emphasis is placed on the integration of disease-oriented and basic science research approaches. The facilities available are those of well-equipped ongoing productive laboratories, in the rich environment of the John Hopkins Medical Institutions.
