A novel therapy for pediatric juvenile spinal muscular atrophy. Pediatric Neurology

      Аннотация: There is no satisfactory therapy for many of the disabling genetic disorders such as pediatric juvenile spinal muscular atrophy (Wohlfart Kugelberg Welander syndrome). The aim of this book is to describe a novel therapy for pediatric juvenile spinal muscular atrophy which resulted in an obvious improvement that has never been reported before. Two unrelated Iraqi boys with pediatric juvenile spinal muscular atrophy were observed, and the one who was more severely affected was treated with a novel therapy which resulted in obvious improvement within less than one month. The less affected boy received no treatment and didn’t show any improvement after few months.

Ключевые слова: juvenile spinal muscular atrophy, Novel treatment