Spinal Muscular Atrophy: Huge Steps

Cerebrum. 2019 Mar 1:2019:cer-03-19. eCollection 2019 Mar-Apr.

Abstract

Spinal muscular atrophy is the number one genetic cause of infant death. Until recently, half the babies born with it would die before their second birthdays, their hearts and lungs becoming too weak to continue. Medical care improved the odds somewhat, but new discoveries and therapeutic developments have improved survival rates significantly-and more good news may be on the horizon.