More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

Months after promising help for “hundreds of thousands of kids,” the FDA approved the decades-old medicine for a disease documented in less than 50 people.

Incubated by Atlas Venture, the biotech startup is advancing a daily pill it sees potentially helping people with dystonia or ...

The Zepbound developer is joining other drugmakers’ rush into China as it plans to boost its oral solid dosage supply chain to support the launch of its obesity pill orforglipron.

The proposed acquisition would hand Servier a couple experimental drugs in human testing as well as Ojemda, an approved ...

Prasad contributed to an uncertain regulatory climate for gene therapy makers focused on rare conditions. Shares of many ...

Company shares surged in after-market trading on results for a drug Vertex acquired in a multibillion-dollar deal and sees as ...

The Kanakee project builds on the company’s efforts to increase its U.S. footprint, where it has already spent more than $3 ...

The therapy, mezigdomide, is part of group of medicines the pharma sees as successors to its lucrative multiple myeloma drugs ...

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

The result “pulls the rug” out from under giredestrant, according to one analyst, sparking another share selloff following disappointing obesity data last week.

Ipsen found cases of secondary malignancies in a confirmatory study of Tavzerik, a medicine it got in a purchase of Epizyme ...