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.

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

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 ...

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 ...

Biogen has partnered with Alcyone Therapeutics to improve the delivery of its spinal muscular atrophy drug Spinraza, handing over $10 million upfront for a global license to an implantable device.

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 ...

Spanish privately-held pharma company entered into an agreement to acquire USA-based TerSera's Infusion Specialty Therapies ...