Respiratory insight to congenital muscular dystrophies and congenital myopathies and its relation to clinical trial

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Congenital muscular dystrophies (CMDs) and congenital myopathies (CMs) constitute the two most important groups of congenital onset muscle diseases. These diseases share features such as early onset of symptoms (in the first year of life), genetic causes, and high risks for restrictive lung disease and orthopedic deformities [1-3]. CMDs are defined as early onset muscle disorders in which the muscle biopsy is compatible with the presence of a dystrophic process without histological evidence of another neuromuscular disease.

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