Positive pre-BLA meeting completed with FDA for z-rostudirsen in exon 51 DMD; on track for BLA submission in Q2 2026 and potential launch in Q1 2027 - - Positive cardiopulmonary results and long-term ...

It is tempting to understand muscle's role in the body as a simple mechanical motor. But the truth is much more complex: our ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS ® ...

If approved, Itvisma (intrathecal onasemnogene?abeparvovec) will be first and only gene replacement therapy for children two years and older, teens and adults with SMA in the European UnionItvisma dem ...

April 13 (Reuters) - Gym operator Basic-Fit (BFIT.AS), opens new tab has suffered a data breach involving around 1 million members with 200,000 in the Netherlands alone, a company spokesperson said on ...

This study reveals abnormal iron accumulation in the muscles of patients with Facioscapulohumeral Muscular Dystrophy (FSHD) and in mice expressing muscle-specific DUX4 (DUX4-Tg mice). Contrary to ...

PCWorld reports that Spotify offers a hidden “Basic” tier launched in mid-2024 for $10.99/month, providing music-only streaming without audiobooks or lossless audio. This plan requires existing ...

Function Health, a startup that offers lab testing and body scans for early disease detection, scored nearly $300 million in series B funding, boosting its valuation to $2.5 billion. Redpoint Ventures ...

Imagine that you’ve never been to Earth. You visit first in winter, where someone introduces you to water. From a glass, they pour it out over your hand. You drink. Remarkable. Imagine that you walk ...

Microsoft open-sourced the MS-BASIC language. Bill Gates would never have seen this coming back in the day. MS-BASIC 1.1 was many developers' first language. In 1976, they rebranded Altair BASIC to ...

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 research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...