In the U.S., Elucirem is indicated in adults and now in pediatric patients including term neonates, for use with magnetic resonance imaging (MRI) to detect and visualize lesions with abnormal ...

Spinraza is a medication used in children (including newborns) and adults to treat spinal muscular atrophy. Treatment begins with four loading doses, followed by one maintenance dose every 4 months.

A retrospective, single-center study out of the Czech Republic tested the safety and efficacy of unilateral interlaminar fenestration (UILF) on the convex side in cases of SMA that also exhibit severe ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

West Palm Beach, Florida — In an interim analysis of an extension study, intrathecal injection of autologous bone marrow–derived mesenchymal stem cells (MSCs) has been associated with favorable ...

The findings, say the researchers, confirm that repeated intrathecal injections of the gene-targeting therapy are safe, adding to the limited data on the long-term effect and safety of the treatment ...