Egypt Fights Spinal Muscular Atrophy
Omar Elsaka, Moneer Ayman Noureldean, Mohamed Adel Gamil, Mostafa Tarek Ghazali, Ashraf Hamada Abd Al-Razik, Dalia Hisham
Asian Journal of Research and Reports in Neurology · pp. 168–170 · Published 21 Dec 2021
Abstract
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.
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