Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.

A positive CHMP opinion for Itvisma is an important step toward addressing this gap." Professor Tim Hagenacker, Department of Neurology, University Hospital Essen, Essen, Germany, said: “Itvisma met ...

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.

Oligonucleotide (oligo) therapies have been in development for decades. Recent advances in molecular design, chemistry, ...

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 ...