The Nature Index 2025 Research Leaders — previously known as Annual Tables — reveal the leading institutions and countries/territories in the natural and health sciences, according to their output in ...

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.

LINCOLNSHIRE, Ill.--(BUSINESS WIRE)--Today, Nexus Pharmaceuticals announced the launch of Baclofen Injection, USP. This sterile solution is designed for intrathecal administration and serves as a ...

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 ...

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 ...

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 ...

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 ...