Neurofilament light (NFL) is a powerful and sensitive biomarker for tracking neuronal health and disease progression.

A pioneering team at St Jude Children's Research Hospital in the US say they successfully treated a child diagnosed with SMA ...

Biogen has bolstered its pipeline by licensing ex-US rights to a drug for Dravet syndrome – a rare form of epilepsy – from ...

In addition to launching TRYNGOLZA, we are pleased that higher dose Nusinersen is one step closer to the market with the recent FDA and EMA acceptances of Biogen's regulatory submissions.

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

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

After hours: February 21 at 6:56:05 PM EST ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

SMA newborn screening programs were available in 31 countries in 2023, covering about 7% of the world's newborns, per a study.

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) reported its fourth-quarter 2024 earnings, revealing a stronger-than-expected financial performance. The company posted an earnings per share (EPS) of -0.66, ...