Spinraza treatment may help restore lipid balance to the cerebrospinal fluid (CSF) of children with SMA, according to a small ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

A UK study found more than 80% of 80 SMA type 1 children on a DMT had scoliosis or other abnormal spinal curvature needing ...

This new formulation provides an additional treatment option for pediatric and adult patients, making the therapy more accessible and convenient in tablet form. Evrysdi, marketed by Genentech, a Roche ...

Royalty also holds rights on Biogen’s MS medication Tysabri (natalizumab) and spinal muscular atrophy drug Spinraza (nusinersen), acquired through agreements with Ionis and Perrigo. The deal coincides ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stock has reached a new 52-week low, touching down at $30.86, with InvestingPro data showing the stock trading at $30.89, significantly below its 52-week high ...

The approval of this new tablet builds on previous successes in SMA treatment, joining the existing oral liquid version of risdiplam (Evrysdi), as well as Biogen's nusinersen (Spinraza) and Novartis ...

FY24 EPS is projected at $16.34. The FDA approved maintenance dosing regimen for Leqembi in Alzheimer's, while Biogen's nusinersen higher-dose regimen is under review. Our government trade tracker ...

The FDA and the European Medicines Agency accepted to review supplemental marketing applications seeking approval for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA).

Submitted apitegromab U.S. Biologics License Application (BLA) for patients with spinal muscular atrophy in January 2025; U.S. launch anticipated in 4Q 2025 Remain on track to submit European Union ...

The trial will evaluate apitegromab in patients who have been or are continuing to be treated with any currently approved therapy such as nusinersen (Spinraza, Biogen) or risdiplam (Evrysdi ...