A group of neuromuscular disorders characterized by progressive muscle weakness resulting from the degeneration and loss of the lower motor neurons.
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A rare, genetic proximal spinal muscular atrophy characterized by degeneration of alpha motor neurons in the anterior horns of the spinal cord and lower brain ...
Cherubism can cause optic atrophy, which is usually due to the mass effect in the optic chiasm or optic nerve by the lesions extending into skull base. Most of ...
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Spinal muscular atrophy (SMA) is a group of inherited neuromuscular disorders characterized by loss of nerve cells in the spinal cord called lower motor neurons ...
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It is defined as a degeneration of the alpha motor neurons from the anterior horn cells in the spinal cord or of the motor nuclei present in the brainstem, the ...
May 26, 2023 · Orphan and rare diseases include more familiar conditions such as cystic fibrosis, Lou Gehrig's disease and Tourette's syndrome.
Muscle atrophy is defined as a decrease in the mass of the muscle and can be a partial or complete wasting away of a muscle.
Jun 17, 2019 · Sika Dunyoh: A rare disease in the United States is defined as a disease that affects less than 200,000 people. The definition of a rare disease ...
Symptoms of some rare diseases may appear at birth or in childhood, including spinal muscular atrophy, lysosomal storage disorders, patent ductus arteriosus ( ...
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce ...