More Hong Kong patients suffering from spinal muscular atrophy (SMA) will be able to enjoy a government subsidy to control ...

Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular ...

New analyses from DEVOTE Part C further characterize the improvements in motor function in participants with SMA who ...

New Data for Nusinersen Underscore Biogen’s Commitment to Advancing Clinical Research to Improve Outcomes in SMA Provided by GlobeNewswire Jun 27, 2025, 2:00:23 PM ...

Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for ...

Nusinersen in SMA: indication of major added benefit in children with early-onset disease Treatment delays permanent ventilation of the children and prolongs life. In addition, the children ...

Nusinersen "alters SMN2 gene splicing to promote the production of full-length SMN protein," write the investigators. In CHERISH, they enrolled children with confirmed 5q SMA who had symptom onset ...

Nusinersen offers only modest, if any, benefit in improving motor function for patients with spinal muscular atrophy (SMA) ― and it's associated with significant risks and adverse events, new ...

Findings from an Italian cohort suggest that nusinersen may benefit fatigue measures in patients with spinal muscular atrophy (SMA) type III; however, drawing definitive conclusions in this area ...

Across the 18 patients with SMA1, nusinersen resulted in a significant reduction in CSF levels of some, though not all, of the neuroinflammatory molecules that were increased at baseline.

Nusinersen is an antisense oligonucleotide drug that modifies pre-mRNA splicing of SMN2 to promote increased production of full-length SMN protein. 11 In a phase 2, open-label, dose-escalation ...