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, ...

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

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 ...

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.

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 ...

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 ...

Investigation Discovery’s The Curious Case Of… The Woman Dying for Attention examines how Sarah Delashmit faked cancer and ...

An assessment of a drug for a rare muscular condition was removed from the website of the National Centre for Pharmacoeconomics, at the request of the Department of Health.

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 ...

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

The Food and Drug Administration’s vaccine chief, Dr. Vinay Prasad, will depart the agency next month, an FDA spokesperson ...