Combining the experimental and approved SMA therapies BIO101 and Spinraza in the lab was found to improve muscle function in ...

Treatment Market is projected to grow at a healthy rate of around 13% by 2026. This growth is primarily driven by the ...

The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys ...

Spinraza (nusinersen) may help stabilize lung function in people with SMA types 2 and 3, delaying the need for breathing ...

The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and changed the indication of the drug after reports of fatal liver injury emerged ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients.

The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating ...

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year ...

New research has demonstrated that a combined regimen of growth hormone and testosterone is safe, well-tolerated, and is ...

Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 ...