The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in ...

The French biotech is developing a new drug approach in a disease market that’s projected to grow to $30 billion by 2030.

George Tidmarsh, a Stanford University physician who founded Horizon Pharma and later ran La Jolla Pharma, was named head of ...

The French pharma will pay $1.15 billion upfront to buy Vicebio and gain access to non-mRNA vaccine candidates against RSV, ...

The $50 billion target, which AstraZeneca plans to meet by 2030, includes new manufacturing projects in Virginia and ...

Leveraging a CDMO’s established platform for AAV manufacturing can significantly streamline gene therapy development.

The startup is using elements of gene and cell therapy to create medicines that could be useful against an array of tough-to-treat solid tumors.

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

A meeting held by the FDA Thursday spotlighted debate over black box warnings that have limited use of hormone treatment for ...

Experts on the panel shared many of the same views around SSRIs, arguing that the risks of the drugs during pregnancy are greater than currently accepted.

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...

Company CEO Doug Ingram said the pause was necessary for Sarepta to maintain a "productive and positive working relationship" ...