On Wednesday, Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of ...

(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal ...

The mother of a four-year-old boy with a rare muscle disease says screening newborn babies for the condition could "save ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

The Key Spinal Muscular Atrophy Companies in the market include - Genentech, F. Hoffmann-La Roche, Scholar Rock, Biohaven/ Bristol-Myers Squibb, NMD Pharma, Novartis, Hoffmann-La Roche, Genentech, Inc ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...

In our pursuit to advance healthcare, some rare diseases still go unnoticed, with most people unaware of their seriousness. One such condition is Spinal Muscular Atrophy (SMA), a genetic disorder ...

Approval is supported by data from the DEVOTE study which showed the benefit of the SPINRAZA 50 mg and 28 mg regimen in both treatment-naïve and ...

Austin, July 25, 2025 (GLOBE NEWSWIRE) -- Spinal Muscular Atrophy Treatment Market Size & Trends: According to SNS Insider, The Spinal Muscular Atrophy Treatment Market was estimated at USD 4.27 ...