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Molecularly targeted therapy for Duchene Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA): Kuwait Experience

Laila Bastaki

Spinal muscle atrophy (SMA) is an autosomal recessive motor neuron disease caused by progressive degeneration of motor neurons in the spinal cord and in specific motor nuclei of the brainstem (cranial nerve nuclei V, VII, IX, and XII). The disease causes the voluntary muscles to become sluggish and wasted. Symptoms of spinal muscle atrophy differ, and may be mild or disabled, but include a muscle weakness which controls movement. Involuntary muscles are not affected, such as those in the heart, the blood vessels and the digestive tract. SMA weakens the muscles that are nearest to the body's core, including the elbows, knees, thighs and upper back. Within the spine (scoliosis) the affected child can develop a curve due to loss of size and strength of the back muscles. SMA development can also affect respiration and chewing, which can endanger the patient's life.