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This is an autologous cell therapy developed by the NeuroGen Brain and Spine Institute for the treatment of limb-girdle muscular dystrophy (LGMD). The therapy involves harvesting mononuclear cells from the patient's own bone marrow (BMMNCs) and administering them via intrathecal and intramuscular injections. The biological mechanism relies on the cells' myogenic and neurogenic potential; once administered, these cells migrate to sites of muscle damage, where they can differentiate into muscle cells and aid in the repair of damaged fibers. This process may also involve the expression of dystrophin and other myoregulatory proteins, potentially halting disease progression and improving functional outcomes when combined with multidisciplinary neurorehabilitation.
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