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Spinal Muscular Atrophy (SMA) is a group of inherited conditions that affect the motor neurons of the spinal cord. Motor neurons are specialized nerve cells that control the muscles used for activities such as breathing, crawling, and walking. In people …

Mucopolysaccharidosis type I (MPS I) is an inherited condition that affects many different parts of the body. It is considered a lysosomal storage disorder because individuals with MPS I have lysosomes (the recycling center of each cell) that cannot break …