What Is SMA Type 1 and How Is It Treated? SMA type 1 is a severe inherited neuromuscular disease that causes rapid loss of motor neurons, leading to profound muscle weakness in infancy. It is caused by missing or nonworking copies of the SMN1 gene. Treatment options now include a one-time gene therapy and two SMN-boosting medicines, and outcomes are significantly better when therapy starts before symptoms appear. What is SMA type 1?

SMA Type 1 can progress fast—but treatment options are changing the story. Learn how early diagnosis and newer therapies, including one-time gene therapy, can make a difference. Have questions? Drop them below. #GeneTherapy #NewbornScreening #SMAType1 #SpinalMuscularAtrophy #Zolgensma