Evaluation of cerebrospinal fluid biomarkers in pediatric patients with spinal muscular atrophy

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder characterised by muscle weakness and muscle atrophy and classified into five known subtypes based on clinical features. The recent development of novel drugs to treat SMA has been encouraging, and nusinersen is the first drug approved to treat SMA.

Source: Brain and Development - October 6, 2022 Category: Neurology Authors: Yoshiyuki Kobayashi, Nobutsune Ishikawa, Yuichi Tateishi, Hiroki Izumo, Syohei Eto, Yuta Eguchi, Satoshi Okada Source Type: research