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

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.

This marks a significant advancement in gene therapy, as this patient is the second adult SMA patient worldwide to be treated ...

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 ...