Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b data further illustrate potential of salanersen in SMA, ...

A new 3D model shows that Duchenne gene therapy can repair muscle fibers but fails to shut down the signals that drive ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...

The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.

Because technology is a part of columnist Shalom Lim's daily life with Duchenne muscular dystrophy, he weighs in on the ...

This Monday 9 March, the nation is invited to celebrate National Meatball Day with purpose, by turning its love of meatballs ...

The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our ...

Researchers developed a gene therapy for LAMA2-related muscular dystrophy that restored muscle and nerve function in mice. One treatment stabilized disease progression and improved strength, even when ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

Q4 2025 . Management View. Curran Simpson, President and CEO, stated, "2026 is set to be a pivotal year for REGENXBIO with great focu ...

For the second time in less than a year, Vinay Prasad, the head of the U.S. Food and Drug Administration’s vaccines unit, is ...