The Phase 3 STEER trial enrolled more than 100 children with SMA type 2, ages 2-17, who were able to sit, but hadn't walked ...

NMD Pharma Announces FDA Orphan Drug Designation Granted to NMD670 for the Treatment of Patients with Charcot-Marie-Tooth Disease This is the 2nd ...

Prior to Saturday’s women’s basketball game between West Virginia and BYU, the WVU women’s basketball team invited a young ...

The top 10 most-read stories published by SMA News Today in 2024 focused on symptoms, treatments, and diagnosis.

More than three years after Novartis’ plan for a speedy approval for intrathecal Zolgensma was thwarted by an FDA requirement ...

Novartis’ Phase III study has demonstrated significant improvement in motor function in children and young adults with spinal ...

Novartis is in the trial phase for a new experimental medicine that has shown promise in improving motor function in children with a form of spinal muscular atrophy, according to a Dec. 30 report from ...

The pharmaceutical industry is buzzing with innovation, driven by the need for new treatments, tackling unmet medical ...

The firm said it plans to share data with regulatory agencies from the Phase III STEER trial after it met its primary endpoint.

The antisense oligonucleotides market is set to grow at a 6.6% CAGR, reaching $5.5 billion by 2033. Expanding applications in treating genetic disorders and cancer drive market growth. Increased R&D ...

Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, ...

Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...