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BA-1049 (also known as NRL-1049) is a selective small molecule inhibitor of Rho kinase 2 (ROCK2), developed for the treatment of cerebral cavernous malformations (CCM), also referred to as cavernous angioma. It is designed to reduce the accumulation of new lesions and alleviate neurological symptoms associated with CCMs by inhibiting ROCK2, which is implicated in vascular permeability and neuroinflammation. Preclinical studies have shown that BA-1049 reduces lesion volume and hemorrhagic transformation in mouse models of CCM, preserves blood-brain barrier integrity, and suppresses seizures. The drug has demonstrated an 86-fold selectivity for ROCK2 over ROCK1, making it highly targeted for central nervous system disorders. In addition to CCMs, preclinical data suggest potential utility in other neurological diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease, Parkinson's disease, multiple sclerosis, Huntington's disease, spinal muscular atrophy (SMA), stroke, traumatic brain injury, optic nerve injury, peripheral nerve injuries, vasospasm after subarachnoid hemorrhage, hereditary hemorrhagic telangiectasia and Behcet's Disease[1][4][5][6][7][8][9].
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