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GABAmide (gamma-amino-butyramide hydrochloride) is a small molecule GABA receptor agonist that targets both GABA-A and GABA-B receptors. It is a primary active metabolite of the anticonvulsant drug progabide. Unlike its parent compound, GABAmide is highly polar and does not readily cross the blood-brain barrier when administered systemically. Consequently, it has been investigated for intrathecal delivery to treat severe spasticity resulting from conditions such as spinal cord injury, multiple sclerosis, traumatic brain injury, stroke, and cerebral palsy. By delivering the drug directly into the cerebrospinal fluid, researchers aimed to achieve potent antispastic effects while minimizing the systemic side effects associated with oral GABAergic agents. Preclinical studies in rat models demonstrated that intrathecal GABAmide could reduce spasticity at significantly lower doses than those required for systemic administration, positioning it as a potential alternative to intrathecal baclofen.
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