Spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality.
Children with type I SMA typically die by the age of 2 years. Recent progress in gene
modification and other innovative therapies suggest that improved outcomes may soon be
forthcoming. In animal models, therapeutic intervention initiated before the loss of motor
neurons alters SMA phenotype and increases lifespan. Presently, supportive care including
respiratory, nutritional, physiatry, and orthopedic management can ameliorate clinical …