Egypt Fights Spinal Muscular Atrophy

SMA (spinal muscular atrophy) is a neuromuscular illness caused by a genetic (inherited) mutation that causes muscles to weaken and waste away. Motor neurons, a type of nerve cell in the spinal cord that controls muscle movement, are lost in people with SMA. Many pharmaceutical companies continue to invest heavily in research into SMA therapies. Many additional prospective medications, such as valproic acid, phenylbutyrate, hydroxyurea, albuterol, gabapentin, riluzole, olesoxime, and rapamycin, have failed to generate adequate outcomes regarding disease progression.