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Kennedy's disease (also known as spinal and bulbar muscular atrophy, bulbo-spinal muscular atrophy, X-linked spinal and bulbar muscular atrophy) is an X-linked recessive disease that affects men. It is caused by mutations in the gene for the androgen receptor. SBMA, spinal and bulbar muscular atrophy. Currently, there is no definitely effective treatment for SBMA; however, some disease-modifying drugs have been tested, based on the results of basic research to elucidate the disease mechanism of SBMA ( table 1 ). Spinal and bulbar muscular atrophy (SBMA) is a hereditary neuromuscular disorder that has several facets from a mechanistic point of view. First, SBMA is one of neurodegenerative disorders including Huntington’s disease, caused by exonic trinucle-otide repeat expansion, and it likely has patho-physiological overlap with other repeat expansion. OBJECTIVE: To test the hypothesis that stimulation of muscle with beta-2 agonists reduces disease manifestations in patients, we investigated efficacy and tolerability of clenbuterol in Spinal and bulbar muscular atrophy (SBMA). BACKGROUND: SBMA is an adult-onset, X-linked, lower motor neuron disease, characterized by slowly progressive muscle weakness and atrophy. Spinal and bulbar muscular atrophy (SBMA) is an adult-onset, X-linked hereditary neuromuscular disease caused by polyglutamine repeat expansion in the androgen receptor gene 1, 2. Objective: To test the efficacy and tolerability of clenbuterol in patients with spinal and bulbar muscular atrophy (SBMA). Methods: Twenty patients with a diagnosis of SBMA were given oral clenbuterol (0. 04 mg/d) for 12 months. The primary efficacy end point was the change from baseline of the walking distance covered in 6 minutes at 12 months. Abstract Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disease caused by polyglutamine (polyQ) expansions in the androgen receptor (AR) gene. Spinal and bulbar muscular atrophy, or Kennedy's disease, is an X-linked motor neuron disease caused by polyglutamine repeat expansion in the androgen receptor. The disease is characterised by weakness, atrophy and fasciculations in the limb and bulbar muscles. Spinal and bulbar muscular atrophy (SBMA) is an X-linked, adult-onset neuromuscular condition caused by an abnormal polyglutamine (polyQ) tract expansion in androgen receptor (AR) protein. SBMA is a disease with high unmet clinical need. Recent studies have shown that mutant AR-altered transcriptional activity is key to disease pathogenesis. Spinal and bulbar muscular atrophy (SBMA) is an X-linked neuromuscular disease characterized by late-onset, progressive degeneration of lower motor neurons and skeletal muscle atrophy

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How does Clenbuterol work to improve motor function in SBMA patients?

Clenbuterol is a beta-2-adrenergic receptor agonist that stimulates muscle growth and improves muscle strength. In SBMA patients, it can improve motor function by increasing the size and number of muscle fibers.

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SBMA is a genetic disorder that affects the muscles of the spinal cord and brainstem, causing muscle weakness and atrophy.

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