nusinersen therapy for pediatric spinal muscular atrophy Study Background Spinal muscular atrophy (SMA) is an inherited motor neuron disease caused by the degeneration of anterior horn cells of the spinal cord and brainstem. SMA primarily affects babies and young children and has an incidence of 1:10,000. It is caused by a homozygous
Pediatrics
pediatric liver transplantation: a new biological abdominal wall
pediatric liver transplantation: a new biological abdominal wall Study Background Liver transplantation (LT) is the only definitive treatment for children with advanced liver disease. Pediatric liver transplantation has evolved significantly over the past few decades, with excellent short-term and long-term survival rates. However, graft matching challenges, especially size-matched donors, remain. Also, in
